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 ...

Introduction Surgical and invasive procedures are widely used in adults with degenerative lumbar spinal stenosis when conservative treatments fail. However, little is known about the comparative ...

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 ...

Early intervention in Spinal Muscular Atrophy is crucial for better outcomes, emphasizing the need for timely treatment to ...

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 ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in utero treatment of SMA ...

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