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WEBSep 18, 2024 · SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve …
End Of An Era: MDA Telethon Ends Historic Run, Urgent Fight for...
WEBMay 6, 2015 · The Muscular Dystrophy Association (MDA) announced May 1 that after careful consideration and analysis of evolving realities pertaining to both television viewing habits and to philanthropic giving, it is time for the organization to discontinue producing and broadcasting the MDA telethon after 58 years on the air, and move beyond its historic Labor Day telethon — most recently named the ...
MD and Your Heart Monitor: 6 Things I Wish I'd Known
WEBJan 23, 2018 · 1. Use lotion to remove adhesive. Lotion is the easiest way to remove the adhesive on your skin left from the stickers that connect to the electrodes. It is also the least irritating for your skin, especially if you have sensitive skin like me. Let the lotion sink into your skin before wiping of the adhesive with some tissue paper.
Genetic Testing and Genetic Counseling - Muscular Dystrophy News
WEBJan 6, 2022 · Muscular dystrophy refers to a group of more than 30 conditions characterized by progressive muscle weakness.. Muscular dystrophies are caused by genetic mutations — errors in the “code” contained in a person’s DNA. Genetic testing, as the term suggests, involves analyzing a person’s DNA in order to detect disease-causing mutations.This type of testing typically is done with cells in ...
Audentes Becomes Astellas Gene Therapies, Will Advance MD …
WEBApr 6, 2021 · Named a Gene Therapy Center of Excellence, it will work to advance the development of potentially life-changing gene therapies for several conditions, including muscular dystrophy (MD). “The field of gene therapy has made significant strides to date, and we believe the future holds even greater opportunity to bring new treatments to a wide range of diseases for patients in need,” Kenji ...
'Ordinary Joe' Actor With MD Brings Life to TV Character With...
WEBDec 7, 2021 · The show’s name may be “Ordinary Joe,” but this NBC drama series is proving extraordinary for its authentic depiction of a character with a disability, portrayed by John Gluck, 13, who has collagen VI muscular dystrophy. The show follows Joe Kimbreau, played by actor James Wolk, in three different dimensions based on one decision he made ...
DMD treatment: Exon-skipping therapies - Muscular Dystrophy …
WEBJun 28, 2023 · Certain regulatory approvals have been granted in the U.S. to four exon-skipping therapies designed to treat amenable patients with Duchenne muscular dystrophy (DMD): Exondys 51, Vyondys 53, Viltepso, and Amondys 45.These accelerated approvals were based on early clinical biomarker data showing the therapies can increase production of dystrophin protein, which implies they are likely to ...
Ullrich Congenital Muscular Dystrophy (UCMD)
WEBUllrich congenital muscular dystrophy (UCMD) is a rare hereditary muscle condition that manifests at birth or a few months after birth. It belongs to a group of disorders called collagen type 6-related myopathies and characterized by abnormalities in collagen type 6, a major protein that supports skeletal muscles.. Causes. UCMD is caused by a mutation in one of three genes that provide ...
What Is Duchenne Muscular Dystrophy (DMD)?
WEBJan 19, 2022 · Duchenne muscular dystrophy is caused by mutations in the DMD gene, which provides cells with instructions for making a protein called dystrophin. Working in a complex with other proteins, dystrophin acts like a “shock absorber” in muscle cells, helping to protect the cells from wear-and-tear during the rigorous processes of muscle movements.
Pivotal Phase 3 Trial of CAP-1002 Therapy for DMD Launching Soon
WEBJan 27, 2022 · A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy CAP-1002 as a treatment for Duchenne muscular dystrophy (DMD), is expected to launch soon. Capricor Therapeutics, the company developing CAP-1002, announced that the U.S. Food and Drug Administration (FDA) has given the ...