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Archives
Category Archives: Muscular Dystrophy Treatment
WVU Medicine Children’s offers first dose of gene therapy for Duchenne muscular dystrophy in state – WV News
Posted: Published on April 20th, 2024
State Alabama Alaska Arizona Arkansas California Colorado Connecticut Delaware Florida Georgia Hawaii Idaho Illinois Indiana Iowa Kansas Kentucky Louisiana Maine Maryland Massachusetts Michigan Minnesota Mississippi Missouri Montana Nebraska Nevada New Hampshire New Jersey New Mexico New York North Carolina North Dakota Ohio Oklahoma Oregon Pennsylvania Rhode Island South Carolina South Dakota Tennessee Texas Utah Vermont Virginia Washington Washington D.C. Continue reading
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2-year EDG-5506 treatment helps preserve motor function in BMD – Muscular Dystrophy News
Posted: Published on April 20th, 2024
Oral treatment withEDG-5506, now known as sevasemten, continued to stabilize muscle function after two years in men with Becker muscular dystrophy(BMD) taking part in a Phase 1 trial. Thats in contrast to the progressive deterioration of motor function seen during the natural course of BMD in the absence of treatment. Continue reading
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6-year-old treated for muscular dystrophy at Chicago children’s hospital – USA TODAY
Posted: Published on April 20th, 2024
usatoday.com wants to ensure the best experience for all of our readers, so we built our site to take advantage of the latest technology, making it faster and easier to use. Continue reading
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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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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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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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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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