The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately measure ...

WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

Correspondence to Dr Jacqueline Josee van Ierssel, Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON K1H 5B2, Canada; JMinnes{at}cheo.on.ca While most adults fully recover from ...

Please provide your email address to receive an email when new articles are posted on . Patients who underwent rotator cuff repair and had better preoperative Western Ontario Rotator Cuff Index and ...

-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and ...

-- Data being presented at the 32nd Annual FSHD Society International Research Congress (IRC); Investor and analyst webcast event today, Monday, June 9 at 8:00 a.m. ET-- Del-brax is the first ...