Category: Statistics

J Orthop Surg Res. 2025 Feb 20;20(1):184. doi: 10.1186/s13018-025-05546-0.

ABSTRACT

BACKGROUND: The arthroscopic treatment of shoulder instability is widely debated in the literature and includes very different surgical techniques. Imaging techniques do not allow an accurate assessment of soft tissue quality. The aim of the study is to demonstrate that correct patient selection and a surgical technique involving anterior and posterior capsular ligament repair with axillary retention may be the most crucial factors for successful arthroscopic treatment.

METHODS: Patients with recurrent shoulder instability who underwent arthroscopic stabilization between 2014 and 2015 and aged between 15 and 25 years, were included. The minimum follow-up was 5 years. Specific exclusion criteria were critical bone loss and patients with genetic connective tissue disorders. Demographics, instability characteristics, surgical procedures, recurrences, both dislocations and subluxations, and surgical revisions were evaluated. The surgical procedure, always performed by the same surgeon, involved the use of a minimum of 3 double-suture fixation devices and repair/tensioning of the axillary hammock. Statistical analysis was based on the non-parametric Kendall statistical model and proportions test, with p < 0.05.

RESULTS: The study population consisted of 44 patients. The first dislocation occurred in 61.4% of cases between the ages of 15 and 19. The number of dislocations prior to surgery was < 3 in 29.5%, between 3 and 9 episodes in 25%, and 10 or more episodes in 45.5%. Defined by patients who had no recurrent dislocations and those whose symptom of instability or subluxation did not require surgical revision, the surgical success rate was 90.9%. During the follow-up period, 4.5% of patients required surgical revision. Patients who required none or one reduction maneuver or had less than three dislocation episodes were statistically correlated with no recurrences after surgery. After the first dislocation, the 88.6% of patients had recurrency. Furthermore, if treated after only one episode, 75% of patients were affected by an isolated capsulolabral injury. Prompt stabilization surgery allows a simpler procedure, due to a reduced bone damage and associated injuries.

CONCLUSIONS: Even in a high-risk age group under 25 years, when the bone defect is subcritical, performing a standardised surgical technique including antero-posterior capsulolabral repair with axillary hammock retensioning demonstrates high success rates. Patients who correlate with the highest chance of success are those who have had less than three dislocation episodes and who have not undergone reduction manoeuvres, confirming the importance of soft tissue quality and thus of early treatment.

TRIAL REGISTRATION: Retrospectively registred.

PMID:39979985 | DOI:10.1186/s13018-025-05546-0

Harm Reduct J. 2025 Feb 20;22(1):21. doi: 10.1186/s12954-025-01171-6.

ABSTRACT

BACKGROUND: People who inject drugs (PWID) are more likely to engage in unsafe sexual behavior placing them at high risk of acquiring HIV and other STIs. This study aims to assess the prevalence and predictors of inconsistent condom use with casual and/or paid sexual partners among PWID in Georgia.

METHODS: Integrated Bio-Behavioral Surveillance Survey was conducted among PWID in seven major cities of Georgia. Study design was cross-sectional with respondent-driven sampling (RDS) methodology. Data collection was carried out through individual face-to-face interviews. Of the 2005 PWID who participated in the study, we analyzed a subsample of 619 (30.9%) who reported having casual and/or paid sexual partners during the last 12 months and described prevalence and predictors of consistent condom use.

RESULTS: Consistent condom use during casual and/or paid sex in past 12 months was reported by 49.4% of respondents. The likelihood of consistent use with casual and/or paid sexual partners was statistically significantly associated with residence, family income, drug use frequency, drug dependence and HIV risk self-perceptions. In multivariable analysis independent predictors of always using condom at casual/paid sex during the last 12 months were place of residence (aOR = 6.4; 95% CI: 3.2-12.7), family income (aOR = 2.1; 95% CI:1.3-3.5) and drug use frequency (aOR = 0.6; 95% CI: 0.4-0.9).

CONCLUSION: The study revealed low prevalence of consistent condom use with casual and/or paid sexual partners among PWID in Georgia. Integration of safe sex educational interventions in harm reduction services may improve the rates of condom use among PWID and should focus PWID with lower socio-economic status and residing outside capital city.

PMID:39979984 | DOI:10.1186/s12954-025-01171-6

Plant Methods. 2025 Feb 21;21(1):25. doi: 10.1186/s13007-025-01345-0.

ABSTRACT

The enhancement of rice quality stands as a pivotal focus in crop breeding research, with spectral analysis-based non-destructive quality assessment emerging as a widely adopted tool in agriculture. A prevalent trend in this field prioritizes the assessment of effectiveness of individual spectral technologies while overlooking the influence of sample type on spectral quality testing outcomes. Thus, the present study employed Microscopic Hyperspectral Imaging, Raman, and Laser-Induced Breakdown Spectroscopy (LIBS) to acquire spectral data from paddy rice, brown rice, polished rice, and rice flour. The data were then modeled and analyzed with respect to the amylopectin and protein contents of the rice samples via regression methods. Correlation analysis revealed varying degrees of correlation, both positive and negative, among the three spectral techniques and the analytes of interest. LIBS and Raman spectroscopy demonstrated stronger correlations with the analytes compared to microscopic hyperspectral imaging. Based on the selected correlation variables, feature screening and regression modeling were conducted. The modeling results indicated that microscopic hyperspectral data modeling yielded the lowest coefficient of determination of R² = 0.2, followed by Raman data modeling result was higher than it, which was about 0.5. The modeling effect of polished rice is the best. LIBS data modeling performed best, with a coefficient of determination of 0.6. The influence of different sample types on the modeling results was less than that of Raman spectroscopy, and modeling results of grains were better. The feature matching analysis of Raman and libs spectroscopy techniques showed that there were spectral variables that could match amylopectin and protein in the features obtained by multiple modeling statistics, but some modeling variables failed to match. LIBS matched more variables than Raman. These findings provide valuable insights into the application effectiveness of different spectral techniques in detecting rice contents across diverse sample types.

PMID:39979983 | DOI:10.1186/s13007-025-01345-0

BMC Psychol. 2025 Feb 20;13(1):143. doi: 10.1186/s40359-025-02441-5.

ABSTRACT

BACKGROUND: Although gratitude is a culturally-sensitive construct, it has yet received limited research attention in Arab countries, hence hindering the understanding of its features, correlates, and cross-cultural specificities. To fill this gap, we sought to examine the psychometric properties of an Arabic translation of the 6-item Gratitude Questionnaire (GQ) in an Arabic-speaking sample of adults from the general population of Lebanon.

METHOD: We conducted a web-based survey including 601 participants (mean age 29.91 ± 12.61, 62.7% females). The forward-backward translation method was used for the translation and adaptation of the GQ-6 into the Arabic language.

RESULTS: Findings indicated that a four-item version of the GQ achieved adequate fit statistics with the removal of the two reverse-scored third and sixth items. We found a McDonald Omega coefficient for the total 4-item GQ (GQ-4) scores of 0.88, thus attesting for the good reliability of the scale. Multiple-group Confirmatory Factor Analysis showed that the scale structure was invariant across male and female respondents at the configural, metric, and scalar levels. Females exhibited significantly higher gratitude scores compared to males. Finally, discriminant validity of the Arabic GQ-4 was evidenced through positive significant correlations with social support levels.

CONCLUSION: The Arabic adaptation of the GQ showed good psychometric qualities, suggesting that it is suitable for measuring people’s disposition toward gratitude in Arab backgrounds. Offering the Arabic GQ-4 as a brief, simple, cost-effective, valid, and reliable measure of gratitude to the Arabic-speaking community could help raise awareness about gratitude as a key component for achieving good mental health and wellbeing in Arab contexts.

PMID:39979974 | DOI:10.1186/s40359-025-02441-5

Scand J Trauma Resusc Emerg Med. 2025 Feb 20;33(1):30. doi: 10.1186/s13049-025-01346-x.

ABSTRACT

BACKGROUND: The purpose of this study was to clarify the potentially life-saving critical interventions performed on children below the age of seven by the physician-manned mobile emergency care unit (MECU) in Odense, Denmark. We investigated critical interventions in relation to morbidity and mortality.

METHODS: A retrospective cohort study of all MECU missions involving children below the age of seven. The study period was from October 1 2007 to December 31 2020. Data sources were the MECU Odense database, the Danish National Patient Registry, and the Danish Civil Registration System. Variables were critical interventions, the severity of injury/illness, MECU on-scene time, in-hospital diagnosis and 7-day, 30-day, and 90-day mortality.

RESULTS: The MECU carried out 4,032 missions to children below 7 years. 88 patients (2.2%) received at least one critical prehospital intervention. Upper airway suction was performed in 39 cases (1.0%), endotracheal intubation (all causes) in 36 cases (0.9%), and intraosseous access in 21 cases (0.5%). General anaesthesia was induced in 29 cases (0.7%). Seventeen patients (0.4%) received cardiopulmonary resuscitation and two patients received manual defibrillation (< 0.1%). 3,278 patients were admitted to the hospital and assigned a diagnosis when discharged. The most common diagnoses were assigned within the International Statistical Classification of Diseases and Related Health Problems 10th Revision Chapter XVIII (Symptoms, signs and abnormal clinical and laboratory findings, not elsewhere classified), which includes febrile convulsions. 1,437 patients (43.8%) were assigned diagnoses within this diagnosis group. The overall 7-day mortality in the cohort was 0.74%, 30-day mortality was 0.82%, and 90-day mortality was 1.02%.

CONCLUSION: Prehospital critical interventions are rarely performed in children under the age of 7 years. The low frequency of these interventions may have implications for maintaining the clinical routine of the prehospital care providers.

PMID:39979973 | DOI:10.1186/s13049-025-01346-x

BMC Musculoskelet Disord. 2025 Feb 20;26(1):175. doi: 10.1186/s12891-025-08381-w.

ABSTRACT

BACKGROUND: Bone lengthening and free vascularized fibula graft (FVFG) represent two prevalent modalities for bone defect repair. The objective of this study was to compare the efficacy disparities between FVFG and single – segmental bone lengthening (BL) when the Orthofix external fixator was utilized in the treatment process.

METHODS: The follow – up data of 72 patients with femoral defects, who received treatment from January 2009 to May 2019, were collected for retrospective analysis. Among them, 60 comparable patients were stratified into two groups: one group of 30 patients received free vascularized fibular graft in combination with Orthofix external fixation (FVFG group), and the other group of 30 patients underwent single – segmental bone lengthening using the Orthofix external fixator (BL group). The specific aspects evaluated encompassed operation time, intraoperative blood loss, postoperative Activities of Daily Living (ADL) score, assessment by the Association for the Study and Application of the Method of Ilizarov (ASAMI), limb length discrepancy (LLD), external fixation index, bone union status, and complications.

RESULTS: 1.Surgical – related indicators: The mean operation time in the FVFG group ( 5.04 ± 1.22 h) was significantly longer than that in the BL group (1.58 ± 0.56 h, p<0.001). The intraoperative blood loss (450 ± 190.73 ml) in the FVFG group was substantially higher than that in the BL group (120 ± 46.23 ml, p<0.01). Although the duration of external fixator use in the FVFG group (16.9 ± 3.52 months) was marginally longer than that in the BL group (15.53 ± 2.09 months), this difference did not reach statistical significance (p=0.073). 2.Postoperative complications: According to the Paley classification system, no significant differences were observed between the two groups in terms of nail infection, nail loosening, limb edema, nonunion (including nonunion in the BL group and fibula nonunion in the FVFG group), and postoperative fractures. All complications were effectively managed through appropriate treatment strategies. 3.Functional evaluation: Based on the ASAMI criteria, the excellent – to – good rate of bone outcomes in the FVFG group was 80.0%, while the BL group demonstrated superior results (p = 0.028). The excellent – to – good rate of limb function in the FVFG group was 36.6%, and there was no significant difference between the two groups (p = 0.537).

CONCLUSION: For the treatment of femoral defects measuring ≥ 6 cm, single – segmental bone lengthening with the Orthofix external fixator exhibited advantages in terms of shorter operation time and reduced blood loss compared to FVFG combined with the Orthofix external fixator. Although other outcomes were comparable between the two groups, FVFG demonstrated broader applicability across diverse bone defect classifications, primarily constrained by the defect length and the vascular condition of the recipient site.

PMID:39979967 | DOI:10.1186/s12891-025-08381-w

BMC Med Educ. 2025 Feb 20;25(1):282. doi: 10.1186/s12909-025-06857-w.

ABSTRACT

BACKGROUND: Medical education relies heavily on anatomy as a fundamental component. Irrespective of their specific field of expertise, it is undeniably essential for doctors to possess a comprehensive grasp of anatomical principles. Teaching and obtaining knowledge of anatomy are highly important and challenging disciplines for both educators and learners. The aim of this study was to assess the perception and attitude towards the methods used for teaching and assessing anatomy among postgraduate students.

METHODS: A descriptive cross-sectional study was conducted on three consecutive cohorts of anatomy master students. The data collection process employed a semi-structured questionnaire, and the data analysis was carried out using the IBM Statistical Programme for Social Sciences version 24 (SPSS 24).

RESULTS: There was a total of 44 participants who filled out the questionnaire. Among them, 90.9% chose dissection room sessions as the most effective approach for attaining the necessary learning outcomes. Practical dissection was found to be the most successful educational method in terms of stimulating attention (81.1%), offering enjoyment (70.5%), boosting information retention (65.9%), and ensuring satisfaction (59.1%). Of the study participants, 72.7% of the participants hold the belief that the practical dissection test is the most efficient method for evaluating the designated learning goals. The utilisation of multiple-choice questions proved to be highly beneficial in facilitating good comprehension of anatomy and served as the optimal means for evaluating one’s knowledge among 63.6% of the students.

CONCLUSIONS: Overall, the majority of students held positive views regarding the efficacy of cadaveric dissections in all their forms for transferring knowledge about anatomy. The most effective evaluation procedures were the utilisation of multiple-choice questions and the implementation of the spotter test.

PMID:39979957 | DOI:10.1186/s12909-025-06857-w

Lung Cancer. 2025 Feb 12;201:108421. doi: 10.1016/j.lungcan.2025.108421. Online ahead of print.

ABSTRACT

BACKGROUND: KRAS G12D and G12C mutations have distinct biological traits influencing treatment response. This study examines real-world demographics, clinical characteristics, and first-line treatment outcomes in metastatic non-small-cell lung cancer (NSCLC) patients with these mutations.

METHODS: This retrospective, multi-institution observational study used data from the AURORA database. Patients aged 18 years or older, diagnosed with metastatic KRAS G12D or G12C NSCLC between January 1, 2010, and April 30, 2024, were included. Descriptive statistics compared patient characteristics, and time-to-event outcomes were assessed using Cox proportional hazards regression.

RESULTS: A total of 298 (216 KRAS G12C and 82 KRAS G12D) patients were included. The KRAS G12D group had a higher proportion of never smokers (15 % vs. 1 %, p < 0.01) and PD-L1 < 1 % (36 % vs. 21 %, p = 0.06). No significant differences were observed in overall survival (OS) (HR 1.09, 95 % CI 0.80-1.48, p = 0.60) or real-world progression-free survival (rwPFS) (HR 1.21, 95 % CI 0.92-1.59, p = 0.18) between mutation groups. In KRAS G12C, monotherapy immunotherapy (HR 0.61, 95 % CI 0.39-0.97, p = 0.04) and chemo-immunotherapy (HR 0.59, 95 % CI 0.37-0.94, p = 0.03) improved OS compared to chemotherapy. For KRAS G12D, neither immunotherapy (HR 0.74, 95 % CI 0.29-1.89, p = 0.53) nor chemo-immunotherapy (HR 0.73, 95 % CI 0.34-1.57, p = 0.42) improved OS compared to chemotherapy alone.

CONCLUSION: KRAS G12C and G12D mutations demonstrate distinct clinical characteristics and treatment responses, with poorer immunotherapy outcomes in KRAS G12D patients. Prospective studies are needed to validate these findings.

PMID:39977966 | DOI:10.1016/j.lungcan.2025.108421

Neurology. 2024 Apr 9;102(7_supplement_1):6874. doi: 10.1212/WNL.0000000000208147. Epub 2024 Apr 9.

ABSTRACT

OBJECTIVE: To characterize early adopters of subcutaneous (SC) neonatal receptor (FcRn) blockers efgartigimod alfa and hyaluronidase (SC efgartigimod) and rozanolixizumab, approved for generalized myasthenia gravis (gMG).

BACKGROUND: Monthly survey fielded by an independent market intelligence agency specializing in tracking neurological therapeutic markets, including benchmarking new product launch metrics.

DESIGN/METHODS: Online survey fielded in October 2023, approximately two months after SC efgartigimod and rozanolixumab availability, among 65 US neurologists and neuromuscular specialists.

RESULTS: 42% of surveyed neurologists reported prescribing either SC efgartigimod, rozanolixizumab, or both products within the first two months of commercial availability (“early adopters”). Early adopters reported significantly higher patient shares for originator FcRn blocker IV efgartigimod than non-adopters (11.3% vs 3.6%). Early adopters were more likely than non-adopters to rate themselves as highly familiar with SC efgartigimod (8.1 for adopters, 6.8 for non-adopters on 10-point scale) and to rate both SC FcRn blockers as a significant advance over other agents to treat gMG (7.6 vs 6.4 [SC efgartigimod], 7.1 vs 5.9 [rozanolixizumab]). They were more likely than non-adopters to view more of their patients as candidates for SC efgartigimod (23.4% vs 14.7%). Early adopters rated market access as significantly better for both SC products than non-adopters (6.8 vs. 4.4; 6.0 vs 4.0). They also were more likely to have a positive perception of launch execution for both SC products (7.7 vs 6.3; 6.7 vs 5.1). gMG patient load and past month sales representative contact did not significantly differ between early adopters and non-prescribers.

CONCLUSIONS: Prior use of IV efgartigmod may have increased comfort with SC FcRn products, leading to faster uptake and more positive perceptions of both brands across degree of advance, market access, and launch execution. Disclosure: Georgiana Kuhlmann has received personal compensation for serving as an employee of Amgen. Georgiana Kuhlmann has stock in Amgen. Dr. Cloud has nothing to disclose.

PMID:39977948 | DOI:10.1212/WNL.0000000000208147

Neurology. 2024 Apr 9;102(7_supplement_1):3348. doi: 10.1212/WNL.0000000000205078. Epub 2024 Apr 9.

ABSTRACT

OBJECTIVE: To analyze within-group effect sizes of cipaglucosidase alfa plus miglustat (cipa+mig) and alglucosidase alfa (alg) for efficacy, quality of life (QoL), and biomarker variables in ERT-experienced patients.

BACKGROUND: The randomized, double-blind PROPEL study (ATB200-03; NCT03729362) compared the efficacy and safety of the investigational two-component enzyme replacement therapy (ERT) cipa+mig with alg plus placebo in adults with late-onset Pompe disease (LOPD); 77% of patients had received ERT with alg before study entry (median ERT duration 7.4 years).

DESIGN/METHODS: We analyzed within-group effect sizes of cipa+mig and alg for outcome variables including motor function, lung function and muscle strength tests; patient-reported outcomes/QoL; and biomarkers in ERT-experienced patients. Standardized within-group effect sizes (Cohen’s d for correlated measurements from baseline to week 52) were calculated by dividing the mean change from baseline by the standard deviation of the difference scores.

RESULTS: ERT-experienced patients remaining on alg (n=30) generally showed worsening (d<-0.2) or stability (-0.2≤d≥+0.2) across most outcomes, while those switching to cipa+mig (n=65) mostly showed improvement (d>0.2) or stability. Patients remaining on alg demonstrated statistically significant within-group worsening for sitting and supine forced vital capacity; slow vital capacity; maximal expiratory pressure; and creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and no significant improvements for any outcomes. Patients switching to cipa+mig did not demonstrate significant within-group worsening for any outcomes and showed significant improvements for 6-minute walk distance (absolute and % predicted); upper, lower and overall manual muscle test; Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue; Physician and Subject Global Impression of Change (five of eight subdomains); and CK and Hex4 levels.

CONCLUSIONS: This analysis shows that ERT-experienced patients with LOPD who switched from alg to cipa+mig treatment achieved improvements in a number of outcomes, highlighting the potential of cipa+mig to become an important treatment option for these patients. Sponsored by Amicus Therapeutics Inc. Disclosure: Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion AstraZeneca Rare Disease. Dr. Kushlaf has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Kushlaf has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Kushlaf has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi Genzyme. Drago Bratkovic has nothing to disclose. Barry Byrne has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for AavantiBio. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Barry Byrne has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex. Barry Byrne has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Barry Byrne has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association. Barry Byrne has received intellectual property interests from a discovery or technology relating to health care. Dr. Claeys has nothing to disclose. Jordi Diaz-Manera has nothing to disclose. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EcoR1 Capital, LLC. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cello Health BioConsulting, previously Defined Health. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RAF-5. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Houston NeuroCare. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Shire. Dr. Dimachkie has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RaPharma/UCB. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. An immediate family member of Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SCHOLAR Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jenssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amazentis / Vandria. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roivant. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SANEM. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Third Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Orphazyme . Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roivant . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Covance / Labcorp. The institution of Dr. Dimachkie has received research support from CSL Behring. The institution of Dr. Dimachkie has received research support from Orphazyme. The institution of Dr. Dimachkie has received research support from FDA-OOPD. The institution of Dr. Dimachkie has received research support from NIH. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Maze Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for JCR Pharmaceutical. Priya Kishnani, MD has received personal compensation in the range of $500,000-$999,999 for serving as a Consultant for Asklepios Biopharmaceutical Inc. (AskBio). Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Baebies, Inc.. The institution of Priya Kishnani, MD has received research support from Sanofi/Genzyme. Priya Kishnani, MD has received research support from Amicus Therapeutics. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care. Mark Roberts has nothing to disclose. Dr. Toscano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Dr. Castelli has received personal compensation for serving as an employee of Amicus Therpeutics. Frederick Holdbrook has received personal compensation for serving as an employee of Amicus Therapeutics Inc.. Frederick Holdbrook has stock in Amicus Therapeutics Inc.. Dr. Das has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Wasfi has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Wasfi has stock in Amicus Therapeutics. Benedikt Schoser has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi Genzyme. Benedikt Schoser has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amicus. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Modis Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/ Ra Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Maze Therapeutics. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for National Institutes of Health. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neuromuscular Disease Foundation. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi-Genzyme. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Spark Therapeutics. The institution of Dr. Mozaffar has received research support from Audentes Therapeutics. The institution of Dr. Mozaffar has received research support from Cartesian. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.

PMID:39977945 | DOI:10.1212/WNL.0000000000205078