-
Novartis pays $12B for Avidity and its muscle-targeting RNA platform
In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity […]
-
Getting Ready for the 2026 MDA Clinical & Scientific Conference
It’s that time of year when we come together to connect, share, learn—while accelerating progress […]
-
RegenXBio completed enrollment in pivotal gene therapy trial; still enrolling the confirmatory trial ages 1 year and above
RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 […]
-
Simply Stated: Updates in Neuropathy Ataxia and Retinitis Pigmentosa (NARP) Syndrome
Neuropathy ataxia and retinitis pigmentosa (NARP) syndrome is a rare, maternally-inherited condition caused by mutations […]
-
My family caregivers are feeling the pressure
I have a growing list of limitations, as a result of my facioscapulohumeral muscular dystrophy […]
-
BBP-418 improves walking ability in LGMD2i patients in Phase 3 trial
Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in […]
-
What to Know About Changes to Food Assistance through SNAP
Funding for the Supplemental Nutrition Assistance Program, which provides food assistance to more than 42 […]
-
Avidity Biosciences to be Acquired by Novartis
Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes […]
-
Avidity Biosciences to be acquired by Novartis
When CureDuchenne invested in Avidity Biosciences in 2018, it was with strong belief in their […]
-
I’m giving everything I have for Duchenne MD — in my own way
We are on the brink of November, and it’s beginning to turn cold here in […]
