Category Archives: Muscular Dystrophy Treatment

Although the road has not been smooth, 2023 was witness to unprecedented progress in the treatment landscape of Duchenne muscular dystrophy (DMD), with the approval of Sarepta Therapeutics delandistrogene moxeparvovec (SRP-9001; Elevidys) earlier this summer.1 Despite the milestone approval, only a small percentage of patients with DMD currently stand to benefit from the therapy. Continue reading →

'I was so lucky!' Gilbert Gottfried's widow pays tribute to Aladdin star one year on from his death   Yakima Herald-Republic Continue reading →

Going beyond customs: The Hindu Editorial on customs exemption for drugs, food imported for treatment of rare diseases   The Hindu Continue reading →

Entrada Therapeutics Appoints Dr. Bernhardt Zeiher to its Board of Directors   Marketscreener.com Continue reading →

What is Duchenne Muscular Dystrophy? Continue reading →

28 Reasons we love Rochester one for every day of the Month of Love   Rochester City Newspaper Continue reading →

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Linda MacMullen (seen here with her support dog, Bailey) is hopeful a new treatment for those living with myasthenia gravis will ultimately lead to more independence for her. Photo by Terry Farrell Courtenay resident Linda MacMullen was diagnosed with myasthenia gravis (MG) in 2001, after 10 years of showing symptoms Continue reading →

Back 06/02/22 8:30 AM EDT CAMBRIDGE, Mass., June 02, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in a fireside chat at the Goldman Sachs 43rd Annual Global Healthcare Conference on Tuesday June 14, 2022 at 10:00 a.m. Continue reading →