Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle muscular dystrophy D1 (LGMD1) by blocking a protein that is overactive in some people with this disease. Inhibiting this protein may be a way of correcting muscle weakness in these…
Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy (DMD) — a move that is being hailed as a positive step toward getting these therapies into clinics. Exonics was co-founded in 2017 by CureDuchenne, a nonprofit organization…
Researchers at the University of California in Los Angeles (UCLA) have been awarded a $175,000 grant from Parent Project Muscular Dystrophy (PPMD) to make CRISPR/Cas9 as safe and effective as possible as a gene therapy tool to treat Duchenne muscular dystrophy (DMD). According to the researchers, their planned CRISPR/Cas9 gene…
Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people with LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). LAMA2 MD, also known as merosin-deficient congenital…
The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as well as the corticosteroid Emflaza (deflazacort; by PTC Therapeutics), for treating…
Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The request is made as a conditional marketing authorization (CMA), which may be granted to medicines whose benefit…
Now that the U.S. Food and Drug Administration (FDA) has approved Zolgensma — the world’s first gene therapy for spinal muscular atrophy (SMA) — experts say a similar gene therapy to cure Duchenne muscular dystrophy isn’t far behind. On May 24, the FDA gave its long-awaited…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
A FinancialForce platform has allowed the Muscular Dystrophy Association (MDA) to enhance fundraising efforts while streamlining accounting activities. Since completing the platform launch and implementing an administrative realignment, the MDA has 40% more time to focus on fundraising strategy, according to a FinancialForce press release. Divisions such…
The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells in patients with Duchenne and Becker muscular dystrophy. “Understanding the immune profile and how it changes through the course of disease can provide…
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