#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.

@mda.org #RDAatMDA

Deramiocel Eases Upper Limb Function Decline in Patients With DMD Patients treated with deramiocel demonstrated a 52% attenuation of disease progression compared with matched external patients.

#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

@mda.org #RDAatMDA

Givinostat Appears to Stabilize Respiratory Function in DMD Patients who received givinostat showed a mean annual decline in forced vital capacity %-predicted of 3.6% before and 3.9% after ambulation loss.

#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

#RDAatMDA @mda.org

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

@mda.org #RDAatMDA #MDAConference

#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: "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

#RDAatMDA @mda.org

#MDAConference #RareDisease #RDAatMDA @mda.org

#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

#RDAatMDA @mda.org

#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.

@mda.org #RDAatMDA #RareDisease

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.

#MDAConference #RDAatMDA

Balloons in yellow and blue around marquee lettering that says 75 MDA.

📢 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!

#RDAatMDA @mda.org