A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

The health regulator has requested an update to technical information on the drug, Spinraza, to be included in the Chemistry ...

A new international study has shown that treating babies with spinal muscular atrophy before symptoms appear can dramatically improve outcomes.Emily ...

The FDA letter requested that a technical information update be included in the Chemistry Manufacturing and Controls (CMC) ...

The complete response letter was issued by the FDA during a routine inspection of a third-party fill-finish facility, not because of an issue with apitegromab.

The FDA re-approved Wellcovorin, a drug from GSK, that the company stopped selling more than 25 years ago, as part of a push from the Trump administration to identify the causes of and potential ...

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 ...

Ten-year-old Sebastian Veiman is racing against time to receive €2.5 million gene therapy that could halt — or even reverse — ...

Nathanael was 11 months old when he was diagnosed - now his family hopes screening will spare others the same wait.

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in ...

For investors, SRRK stock offers a high-probability FDA catalyst, meaningful upside in SMA, and additional optionality in obesity and more.