Roll" event kicked off Saturday morning at Xavier University, drawing participants to raise awareness and support for those ...

Spinraza (nusinersen) treatment helped school-age children with SMA be more independent in tasks involving their arms and ...

A Co Down woman is encouraging people living with a muscle wasting or weakening condition, their families and carers to join ...

The Information Day takes place on Wednesday 4 June 2025, in Lisburn, Co. Antrim, and is organised by Muscular Dystrophy UK, ...

Scientists working at the NIH’s BRAIN Initiative have created new viral vectors that one day could lead to gene therapies for ...

The SBMA therapy AJ201 showed good safety, hints of efficacy, and improved physical activity for this SMA type in a trial, ...

Annji Pharmaceutical Co. Ltd.’s rosolutamide (AJ-201, JM-17) achieved positive results in a phase I/II trial in adults with spinal and bulbar muscular atrophy, also known as Kennedy’s disease.

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for ...

Subjects receiving the therapy demonstrated clinically meaningful enhancements in physical and muscle function.

AnnJi Pharmaceutical Co., Ltd., a clinical-stage Taiwanese biotechnology company focused on addressing unmet medical needs in dermatology, neurology, and rare diseases, today announced positive ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy ... even some adults with ...