PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

Precision’s two lead programs, PBGENE-HBV, for chronic Hepatitis B, and PBGENE-DMD, for Duchenne muscular dystrophy, are focused on areas with large patient populations with high unmet need.

Collaboration Revenue: Collaboration revenue was $2.0 million for the second quarter of 2025, compared to $94.7 million for the same period in 2024. This decrease is primarily attributable to the ...

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

Expected cash runway extended into Q3 2027, beyond multiple potential inflection points including Dyne's first planned commercial launch in early 2027 - - Registrational Expansion Cohort of DELIVER ...

Dyne also reported new positive long-term data from adult DM1 patients enrolled in the randomized, placebo-controlled multiple ascending dose (MAD) portion of the DYNE-101 ACHIEVE trial, including ...

European pharma companies are breathing a sigh of relief after the Trump administration posed a 15% tariff, rather than 200% ...

WVE-006, the leading GalNAc-RNA editing oligonucleotide designed to correct AAT protein and address both lung and liver ...

Instead of turning inward after the death of his son, Dr. Greg Gulbransen turned outward: toward documentary photography and people whose lives he might be able to save.

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.