Hansa Biosciences’ Idefirix (imlifidase) has reduced patients’ antibodies to a level that allows dosing in patients with ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from ...

Exploring the impact and challenges of the 'Right to Try' legislation for experimental treatments.

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

At the 2025 Alzheimer’s Association International Conference, one of the bigger splashes was made by an experimental cardiovascular drug. Newamsterdam Pharma Co. NV presented data showing that its ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

A controversial hematologist-oncologist who joined FDA this spring and quickly became its top regulator has left the ...

In the latest episode of STATus Report, Alex Hogan breaks down the confusing, heartbreaking saga of Sarepta and its gene ...

In this week’s edition of InnovationRx, we look at Halle Berry’s menopause startup, Ambience Healthcare’s $243 million ...