Jennifer Goldstein is a specialist in Duchenne muscular dystrophy at the University of Rochester Medical Center’s Center for Health + Technology.

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#MDAConference: "Really the relative risk versus benefits in older DMD patients with preexisting cardiomyopathy or history of heart failure is currently unknown."
-Beth Kaufman, MD, Director, Pediatric Cardiomyopathy Program at Stanford Medicine Children's Health

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#MDAConference: We spoke with Chris Lewis, son of world-famous comedian Jerry Lewis, who through his annual telethons helped raise $2.5 billion for @mda.org

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#MDAConference: "It's important that we're empowering our patients through education and self-management strategies." -Constance de Monts, PT, DPT Neuromuscular Clinical Research Manager/Evaluator at @stanfordmedicine.bsky.social.

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The winner of the first ever @mda.org Legacy Award for Community Impact in Research goes to Donovan Decker, a South Dakota patient and advocate with limb-girdle muscular dystrophy.

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#MDAConference: Katherine Mathews, MD, director of the Iowa Neuromuscular Program wins the 2025 MDA Legacy Award for her contributions to the understanding and treatment of #GeneticDisorders affecting the neuromuscular system.

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#mdaconference: “Medicine as a business is winning out right now over medicine as a profession—not just doctors but all the clinicians involved in multidisciplinary care … I hope I’m wrong.”
- Former FDA Commissioner Robert M. Califf

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📢 Rare Disease Advisor is live at the #MDAConference in Dallas, Texas! 🎉

We’ll be covering key sessions, sharing expert insights, and posting live updates straight from the event. Follow along for the latest in neuromuscular disease research, treatments, and advocacy!

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#MDAConference: Boys with Duchenne muscular dystrophy treated with #givinostat and steroids had less decline in respiratory function after losing ambulation than those treated with steroids alone.

Read more: bit.ly/3FEOFTK

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#MDAConference: Study participants w/ Duchenne muscular dystrophy treated with the investigative cell therapy #deramiocel showed a decreased annual decline in upper limb function over a 5-year period that included a 12-month gap period with no treatment

Read more: bit.ly/3XXGU1e

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