The project addresses a long-standing gap in the field: the lack of a clear human reference standard for muscle regeneration. A new research collaboration between muscular dystrophy organizations aims ...

Obstructive sleep apnea prevalence increased in children with achondroplasia beginning at school age (6-12 years) and remained elevated into adolescence. Recurrent otitis media may accompany ...

Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...

Life expectancy in DMD has improved over time. Previous estimates pointed to a life expectancy of 25 years, with more recent data reporting a life expectancy of 31.7 years (95% CI, 27.4-36.0). 3,4 A ...

Patients with MG who took C5 inhibitors saw greater reductions in concomitant OCS and NSIST use than did those who took efgartigimod. Rare Disease Advisor, a trusted source of medical news and feature ...

At present, no validated tools or scoring systems have been developed to predict prognosis or clinical course of warm autoimmune hemolytic anemia (AIHA). Several potential prognostic risk factors that ...

Spesolimab is a humanized monoclonal immunoglobulin G1 antibody and is the first therapy to target the IL-36 pathway, a key driver of GPP disease processes. Spesolimab has an acceptable safety profile ...

ACTUS-101, AskBio’s ongoing trial of a different gene therapy candidate, will continue with existing participants. AB-1009, an adeno-associated virus-based investigational gene therapy for late-onset ...

The authors argue that the long-held label of "uniformly lethal" may no longer be appropriate and can inadvertently bias clinical decision-making. Homozygous achondroplasia is traditionally ...

Ambulatory boys with DMD treated with delandistrogene moxeparvovec maintained North Star Ambulatory Assessment scores above baseline at year 3. Sarepta Therapeutics announced positive 3-year ...

During consultation, physician-perceived stress showed a nonsignificant positive association with physiologic stress measures only in women with HAE. A comparison of ANS activity during experimentally ...

Efgartigimod improved MG-ADL scores in patients with anti-MuSK antibody-positive, anti-LRP4 antibody-positive, or triple seronegative MG. The US Food and Drug Administration (FDA) has accepted a ...