The mother of a teenage boy, with muscular dystrophy, said her son hasn’t been able to leave their Oakland home for almost a ...

Augustine Therapeutics has raised €78 million ($85 million) in first-round funding for a pipeline headed by a candidate for ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship gene therapy, ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

Bipolar disorder is characterized by extreme mood swings, bouts of mania or hypomania, and episodes of depression.  It is a highly heritable and serious condition, that when untreated comes with a ...

Sarepta Therapeutics (SRPT) saw its shares surge in the last session with trading volume being higher than average. The latest trend in earnings estimate revisions could translate into further price ...

FDA & EMA approvals for rare genetic diseases are fast and require clinical studies with small number of patients Ramat Gan, Israel, March ...

FDA & EMA approvals for rare genetic diseases are fast and require clinical studies with small number of patients Ramat Gan, Israel, March 19, 2025 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE Am ...

July 17, 2024 — A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic ...

The new findings center on a subset of 22 participants who received amyloid-removing drugs the longest, on average eight ...

A cholesterol receptor protein called LDLR appears to be a key driver of neurotoxicity in sporadic ALS, per new research ...