News

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 Biosciences, to develop several promising experimental therapies for various types of muscular dystrophy. The core of the acquisition is Avidity’s antibody-oligonucleotide conjugate (AOC) platform. This innovative technology attaches a small piece of…

DMD treatment Agamree may avoid issues of traditional steroids

Data from two clinical trials confirm that Agamree (vamorolone), an approved anti-inflammatory treatment for people with Duchenne muscular dystrophy (DMD), blocks the action of a receptor involved in regulating the balance of salt and water in the body, and may have advantages over traditional corticosteroids. A study found…

2026 MDA conference to focus on ‘innovation driving us forward’

The Muscular Dystrophy Association (MDA) has announced the agenda for its 2026 MDA Clinical & Scientific Conference — slated for March 8-11 in Orlando, Florida — and the focus, the nonprofit says, is on “groundbreaking research and clinical achievements.” The annual event, held in a different locale each…

DMD gene therapy leads to sustained benefits for 3 boys in trial

Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European…

Agamree becomes 1st approved DMD treatment in Canada

Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada. Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S., the U.K.,…

Exon-skipping therapy shows promise for DMD44

Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730)…