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WEB6 days ago · 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 ...
'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 ...
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.
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 ...
1st Duchenne MD Corticosteroid Treatment, Emflaza, Approved by …
WEBFeb 10, 2017 · by Özge Özkaya, PhD February 10, 2017. A first corticosteroid treatment for Duchenne muscular dystrophy (DMD) regardless of mutation, Emflaza (deflazacort), has been approved for use in patients ages 5 and older by U.S. Food and Drug Administration (FDA). “We are in a new era in the treatment of Duchenne muscular dystrophy.
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 ...
Life Expectancy Increasing for DMD Patients, 40-year Analysis Finds
WEBFeb 1, 2022 · by Yedida Y Bogachkov PhD February 1, 2022. Life expectancy has been increasing in patients with Duchenne muscular dystrophy (DMD), according to an analysis of some 40 years of data, spanning births from before 1970 to the late 2000s. In fact, the median life expectancy of males with DMD born in …
FDA Awards $1M to Duchenne MD Phase 2 Study of Ifetroban in...
WEBOct 2, 2019 · The award marks the first time the FDA has used its orphan drug grant program to support a DMD study, the company said in a press release. The Phase 2 study (NCT03340675), being launched in collaboration with the Vanderbilt University Medical Center, plans to enroll 48 DMD patients, age 7 and older, with evident damage to the myocardium (the heart’s muscular wall).