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Archives
Category Archives: Muscular Dystrophy Treatment
AGAMREE: A Promising Treatment for Duchenne Muscular Dystrophy – Medriva
Posted: Published on February 20th, 2024
Duchenne muscular dystrophy (DMD) is a debilitating condition that predominantly affects males, causing progressive muscle degeneration and weakness. Continue reading
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Muscular Dystrophy: Symptoms, Types, Causes, and Treatment – Metropolis Healthcare
Posted: Published on February 20th, 2024
What is Muscular Dystrophy? Muscular dystrophy comprises a group of conditions causing progressive muscle weakness and reduced flexibility Continue reading
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Reassessing the Process of Drug Development for Duchenne Muscular Dystrophy: Dongzhe Hong, PhD – Neurology Live
Posted: Published on February 20th, 2024
WATCH TIME: 3 minutes "One possibility is that prescribing physicians arent aware of the age range that received the drug in its pivotal trials or have knowledge on the FDA-approval process. When patients receive the drug at a later stage of their DMD, it might impact the predicted effectiveness of the medications." For years, Duchenne muscular dystrophy (DMD) was mainly treated with a combination of corticosteroids, assistive devices, and cardiac and respiratory support until 2016, when the exon-skipping therapies began to emerge. The first approved therapy was eteplirsen (Exondys 51; Sarepta Therapeutics), an antisense oligonucleotide designed to increase dystrophin, followed by other agents, which include golodirsen, viltolarsen, and casimersen. Continue reading
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Revolutionary Therapies Offer Hope to Patients with Neuromuscular Diseases – InventUM – University of Miami
Posted: Published on January 27th, 2024
By: Josh Baxt | January 23, 2024 | 7 min. read| Share Article Summary Amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA) are devastating disorders that affect nerve and muscle function. Until recently, they have been difficult, if not impossible, to treat. Continue reading
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Unbiased assessment of vamorolone for treating Duchenne muscular dystrophy – News-Medical.Net
Posted: Published on January 27th, 2024
The drug vamorolone (Agamree) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). Continue reading
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Phase 3 trial of SRP-9003 gene therapy in LGMD2E starts screening – Muscular Dystrophy News
Posted: Published on January 27th, 2024
Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003in children withlimb-girdle muscular dystrophy type 2E(LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without the ability to walk independently. Continue reading
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Duchenne Muscular Dystrophy Market to Surpass USD 11.7 Bn by 2033, with Enhanced Diagnosis | Marketresearch … – GlobeNewswire
Posted: Published on January 27th, 2024
New York, Jan. Continue reading
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FDAs Marks Advocates for Flexibility in Rare Disease Gene Therapy Trials – BioSpace
Posted: Published on January 27th, 2024
Pictured: Randomized data over a strand of DNA/Nicole Bean for BioSpace Rare diseases, by their very nature, dont fit the moldso neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, argued that non-randomized, single-arm trials could be the best option when testing certain gene therapies for rare diseases Continue reading
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Sharon Hesterlee of the Muscular Dystrophy Association Speaks on New Therapy, Vamorolone – Applied Clinical Trials Online
Posted: Published on December 23rd, 2023
Applied Clinical Trials: What sets Vamorolone apart from other therapies in this market? Sharon Hesterlee: Vamorolone is a little bit different because we've heard a lot about gene therapies and some pretty high tech, very specific, genetically focused medicines in the Duchenne space Continue reading
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