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 ...

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 ...

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.

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, ...

From the outside, Savannah McKell, from NSW, seemed to have a picture-perfect life. But behind closed doors, the Newcastle ...

Billionaire Ty Warner has donated $5 million to complete funding for the massive renovation of Dwight Murphy Park in Santa Barbara. “This is more than a ...

Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") announced that a new indication application (the "Application") for the Company's trophoblast cell-surface antigen 2 (TROP2)-directed ...

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