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

The Information Day takes place on Wednesday 4 June 2025, in Lisburn, Co. Antrim, and is organised by Muscular Dystrophy UK, the leading charity for more than ...

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

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 ...

Finally, young children often didn’t open their mouths when asked; the authors point out that if this study is repeated with older children or adults ... of spinal muscular atrophy may differ ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for ...

Michela Luciano is a Science Writer at BioNews. Originally from the picturesque mountain town of L’Aquila, Italy, Michela ...

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 ...