October 2025

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 RNA (an oligonucleotide) to an antibody. Essentially, the…

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It’s that time of year when we come together to connect, share, learn—while accelerating progress in neuromuscular (NMD) healthcare and scientific progress. This conference is one of the most meaningful gatherings in the (NMD) community—bringing together clinicians, researchers, industry partners, advocates and individuals living with NMD. The four-day conference promises rich content, networking, and collaboration…

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RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial, with topline data expected in Q2 2026.  They are continuing to enroll participants aged 1 year and above in the confirmatory trial, and have initiated manufacturing of commercial supply of drug. Read more here: https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-completion-pivotal-enrollment-and-initiates The…

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Neuropathy ataxia and retinitis pigmentosa (NARP) syndrome is a rare, maternally-inherited condition caused by mutations in mitochondrial DNA, affecting the energy-producing structures within cells. The condition typically begins in childhood or early adulthood and presents with a wide range of symptoms. These may include learning difficulties, muscle weakness (particularly around the eyes), uncoordinated movements (ataxia),…

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I have a growing list of limitations, as a result of my facioscapulohumeral muscular dystrophy (FSHD), my blindness, and other injuries from a long-ago head-on collision with a drunk driver. My wife, Wendy, and my daughter Jill, who are my primary caregivers, provide essential help every day: preparing meals, doing the laundry, and much more.…

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Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular dystrophy type 2i (LGMD2i) — characterized by muscle wasting at the shoulders and the hips — in a Phase 3 clinical trial, according to new interim data announced by developer Bridgebio Pharma. The experimental therapy was…

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Funding for the Supplemental Nutrition Assistance Program, which provides food assistance to more than 42 million children and adults across the country – including 4 million people with disabilities – will run out in November as a result of the federal government shutdown. Unless the administration takes emergency measures, like it did with the Special…

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Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes Avidity’s late-stage programs, including delpacibart zotadirsen (del-zota) for Duchenne. According to Avidity’s announcement, the company and Novartis intend to keep ongoing studies running smoothly and plan for a seamless transition of programs. The acquisition is expected to close in the…

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When CureDuchenne invested in Avidity Biosciences in 2018, it was with strong belief in their technology and the promise of what it might be able to achieve in Duchenne.  We congratulate Avidity on all their success to date, and celebrate with them on this important new development.  And we look forward to working with Novartis…

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We are on the brink of November, and it’s beginning to turn cold here in Nebraska. My husband and I are debating whether we should turn the heat on. I say yes. He says no. Each morning before school is a frenzy as we search for jackets our children can’t remember where they put. My…

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