A decentralized platform connecting researchers to coded data and samples from people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) was presented in a poster at the National Organization for Rare Disorders (NORD) Breakthrough Summit 2021. The platform, called CureDuchenne Link, is a data hub that connects the information provided by DMD and…
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is well tolerated and leads to an overall improvement in motor ability compared to baseline in patients with Duchenne muscular dystrophy (DMD), according to results from the SRP-9001-101 study reported at the World Muscle Society (WMS) 2021 Virtual Congress. These improvements were maintained over 3 years, meaning the response was durable. Roche and…
Fenofibrate showed therapeutic effects in Duchenne muscular dystrophy (DMD) models, according to a new study published in the British Journal of Pharmacology. During the study, treatment with fenofibrate improved muscle function and reduced muscle damage in mdx mice, the most common DMD animal model. The study showed that fenofibrate inhibited the expression of myostatin protein…
By 2025, between 10 and 20 new gene therapies could win approval annually by the US Food and Drug Administration (FDA), most of them delivered via adeno-associated virus (AAV). Yet Duchenne muscular dystrophy (DMD) presents some unique challenges for AAV-delivered gene therapy — namely because the dystrophin gene, which is missing in DMD, is one of…
Don’t overlook cognitive and behavioral issues linked to Duchenne muscular dystrophy, said participants in an online session hosted by CureDuchenne.
Caregivers of patients with DMD often neglect their own sleep, emotional well-being, and happiness, experts warned in a CureDuchenne Futures session.
Six biotech companies working toward a cure for Duchenne muscular dystrophy summarized their progress during an online conference session on exon skipping.
The session was an informal chat where the moderators and panelists talked about what it is like to live with a rare disease like DMD.
Anti-ADAMTS1 antibody relieved muscle dysfunction and fibrosis in a mouse model with Duchenne muscular dystrophy (DMD), found in a new study by Chinese researchers published in the journal Life Sciences. This finding suggests ADAMTS1 could be a new therapeutic target for the disease. ADAMTS1 is a metalloproteinase that was reported to be highly expressed in…
Researchers have developed homology-independent targeted integration (HITI)-mediated gene editing to correct full-length human dystrophin in Duchenne muscular dystrophy (DMD). The authors of this new method observed full-length dystrophin restoration in skeletal and cardiac muscles of a humanized mouse model of DMD, the hDMDΔ52/mdx model, recently published in Molecular Therapy. The mice contained a full-length human…
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