The denial sets back a drug Scholar Rock hopes to become part of a new standard of care for spinal muscular atrophy, treatment for which has changed dramatically over the last decade.

The U.S. Food and Drug Administration's so-called complete response letter is related to issues identified during a routine ...

Scholar Rock Holding said the Food and Drug Administration has turned away its application seeking approval of apitegromab for the rare genetic neuromuscular disease spinal muscular atrophy due to ...

Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for ...

The field of immunology has witnessed significant expansion, delving into novel antibodies and their associations with various diseases. This growth has been mirrored in neuromuscular medicine, ...

An international team of researchers led by Duke-NUS has identified rare mutations in the SPNS1 gene as the cause of a previously unrecognized multi-organ disorder. A research team led by Duke-NUS ...

Every movement we make, whether walking, speaking or even breathing, depends on the health of our muscles and nerves.

Salk Institute researchers identify dozens of microproteins involved in fat storage, offering new potential drug targets for future obesity therapeutics LA JOLLA (August 7, 2025)—The obesity rate has ...

At least three people have died in a Legionnaires’ disease outbreak in Harlem, authorities said in the most recent update, in the latest summer flare-up of the disease that officials say primarily ...

Justin Timberlake recently shared his Lyme disease diagnosis on social media, calling the experience "relentlessly debilitating." "If you've experienced this disease or know someone who has — then you ...

Ozzy Osbourne’s death this week at the age of 76 has ignited curiosity about his Parkinson’s disease and how it contributed to his death. Parkinson’s, which affects memory, thinking and movement, is ...