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.

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

Cell and gene therapy (C&GT) is set to revolutionise treatment of complex diseases, offering new possibilities for patients.

The National University Centre for Organ Transplantation (NUCOT) has launched the inaugural Tree Planting Legacy Project, ...

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

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

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 ...

Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory ...