May 20th 2024
A new report says early evidence of using gene therapy to treat monogenic diseases offers reason for optimism.
Case Report of Patient Death Following High-Dose Gene Therapy for DMD
October 19th 2023In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.
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Avidity Biosciences Gains FDA Orphan Drug Designation for Patients With DMD
October 9th 2023Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).
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Researchers Highlight Restrictive Coverage of Gene, Cell Therapies
June 28th 2023The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations.
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FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy
June 22nd 2023The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.
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