Congratulations to Marwa Zafarullah, MS, PhD, for presenting her amazing research in Dr. John Day's Lab at the MDA Conference 2026 in Orlando (Mar 8–11). The meeting drew 2,366 attendees from 44 countries and highlighted advances in neuromuscular science and care. #MDAConference #Neuromuscular

Women carriers of Duchenne or Becker muscular dystrophy with late gadolinium enhancement on cardiac MRI had significantly higher fatigue scores in a retrospective cohort study presented at the 2026 #MDAconference.

Read more on NeurologyLive❗

#Neurology #Cardiology #MuscularDystrophy

#MDAConference: Anjan Aralihalli, Founder of Raya Therapeutic, Inc., and Member of the Board of Directors at Muscular Dystrophy Association speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

Watch the interview here 🎥

#RareDisease #MedSky

#MDAConference: Barry Byrne, MD, PhD, Gene therapy expert and Board Member of the @mda.org, speaking at the MDA’s 2026 Clinical & Scientific Conference in Orlando, FL.

Watch the interview here 🎥

#RareDisease #MuscularDystrophy #GeneTherapy

#MDAConference: Lily Sander, 18, @mda.org’s 2026 National Ambassador, who introduced Allison Moore, Founder and CEO of the Hereditary Neuropathy Foundation, and winner of the 2026 MDA Legacy Award for Achievement in Clinical Research.

Watch the video here 🎥

#RareDisease #MedSky

#MDAConference: Sharon Hesterlee, MD, President and CEO of the Muscular Dystrophy Association, opening the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

Watch the video here 🎥

#RareDisease #MedSky #MuscularDystrophy

The Medlive team recently shared research at the #MDAconference with our poster on advancing clinician education in #BeckerMuscularDystrophy. Developed with @nordrare.bsky.social & MDA, this #CME initiative explores improving clinician awareness & preparedness for emerging therapies.

bit.ly/47u8LuJ

🧬 Had a fantastic time at the #MDA Conference in Orlando! I shared some of our work on titin (TTN). Great discussions and new ideas sparked with colleagues working on #titin related myopathies. Excited for what comes next! 💪 #MDAConference @myofinlab.bsky.social @folkhalsanresearch.bsky.social

The 16 Minute Challenge: Connecting With Patients in Limited Time Dr. Gramszlo encouraged clinicians to embrace silence rather than rushing to start a new conversation or feeling uncomfortable with it.

#MDAConference: Connecting with patients is crucial and strategic despite the severely limited time available during a visit, according to presenters at the 2026 @mda.org Clinical and Scientific Conference.

Read here: https://bit.ly/4sC97rc

#RareDisease #MuscularDystrophy

#MDAConference: Craig McDonald, MD, Director of the Neuromuscular Disease Clinic at @ucdavishealth.bsky.social, speaking about @sarepta.com's gene therapy delandistrogene moxeparvovec-rokl (#Elevidys) for #Duchenne at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#MDAConference: Barry Byrne, MD, PhD, Gene therapy expert and Board Member of the @mda.org, speaking at the MDA’s 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #Duchenne

Ethical Challenges in Newborn Screening Remain Unresolved As technology advances, questions arise regarding how extensive newborn screening should be.

#MDAConference: Although a newborn screening program exists in every US state, many ethical concerns raised by them remain unresolved, experts said at the 2026 @mda.org Clinical and Scientific Conference.

Learn more: https://bit.ly/3P4wUSn

#RareDisease #NewbornScreening

#MDAConference: Mark Awadalla, Chief Development Officer at Capricor Therapeutics, speaks with Rare Disease Advisor at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL, about #Deramiocel—an investigational, allogenic cell therapy to treat #DMD

Watch the interview here 🎥

Corticosteroid Regimens Evolve While Remaining Central to DMD Treatment "Steroids remain the cornerstone of treatment despite all of the exciting treatments," Dr. Saylam said.

#MDAConference: Corticosteroid treatment for Duchenne muscular dystrophy (DMD) continues to evolve, as new regimens show significant benefits, an expert reported at the 2026 @mda.org Clinical and Scientific Conference.

Read more: https://bit.ly/4s36M8S

#RareDisease #DuchenneMuscularDystrophy

#MDAConference: Natalie Truba, PhD, Clinical Psychologist at @nationwidekids.bsky.social in Columbus, Ohio, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #MedSky

#MDAConference: Kathryn Wagner, MD, PhD, Professor Emerita of the Johns Hopkins University School of Medicine in Baltimore, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy

#MDAConference: Mark Awadalla, Chief Development Officer at Capricor Therapeutics, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL, about deramiocel—an investigational, allogenic cell therapy to treat DMD.

#RareDisease #MuscularDystrophy #Deramiocel #Duchenne

🧬 Deramiocel is back under #FDA review for #DMD cardiomyopathy.

The agency lifted a prior CRL and set a new PDUFA date of August 22, 2026, continuing evaluation of Capricor’s cell therapy following new #Phase3 HOPE-3 data.

Visit the NeurologyLive website to read more‼️
#Neurology #MDAconference

Noncanonical LeuRS Function Offers New Avenue for DMD Treatment "Inhibiting LeuRS may not directly affect muscle regeneration but rather reduce degeneration through restored autophagy," Dr. Chen said.

#MDAConference: Leucyl-tRNA synthetase (#LeuRS) may represent a new therapeutic target for improving muscle quality and strength in Duchenne muscular dystrophy (#DMD) through its noncanonical function in autophagy regulation. @mda.org

Read more: https://bit.ly/3MSNKTG

FDA Addresses Criticism Over Inconsistent Evaluations of Rare Disease Trial Endpoints Due to differences in the nature of products overseen by CDER and CBER, they may vary in their approval of the same trial endpoint.

#MDAConference: The @fda.gov faces criticism over inconsistencies in trial evaluations for rare disease therapies across its 2 divisions, the Center for Drug Evaluations and Research and the Center for Biologics Evaluation and Research. @mda.org

Read here: https://bit.ly/4by56hU

Clinical Trials Exploring Multiple Frataxin Restoration Strategies in Friedreich Ataxia Three clinical trials are investigating various approaches to restoring frataxin.

#MDAConference: Ongoing trials are investigating the potential of restoring the #Frataxin protein in individuals with #FriedreichAtaxia (FA), despite the challenges this therapeutic approach presents. @mda.org

Read more: https://bit.ly/4ukONwp

✨ Exciting news! Our editorial team is a Northeast regional finalist in the 2026 Azbee Awards for their 2025 coverage of the #MDAConference.

👏 Larry Luxner, Brian Murphy, Riya Ajmera, Stephan Cho, Terri Airov, Michael Nace

Check out our 2026 Coverage: https://bit.ly/4roNTMD

This image features a quote from Edward Neilan, Chief Medical and Scientific Officer at NORD, discussing the importance of relevant and reliable real-world data for regulators like the FDA. It mentions AI's potential role in processing large data amounts. The background is a light blue graphic design. The bottom right corner displays the Rare Disease Advisor logo.

#MDAConference: Edward Neilan, Chief Medical and Scientific Officer at @nordrare.bsky.social, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #AI #MedTech

The image features a quote about the impact of AI on clinical development, emphasizing patient-centric care and reduced administrative burden. There is a picture of a person identified as Tim Miller, MD, Global Head at ThermoFisher Scientific. The background includes the Rare Disease Advisor logo and the hashtag #MDAConference.

#MDAConference: Tim Miller, MD, Global Head of Scientific and Therapeutic Development at @thermofishersci.bsky.social, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #AI

The image features a quote in a blue circle with rounded corners at the top left. The quote discusses the obesity pandemic in the US starting in the 1980s and recent advancements in weight-loss medications. To the right, there is a person labeled as Jamie Moore, MD, Medical Director of the Pediatric Obesity Program at Children's Hospital Colorado, standing against a neutral blurred background. The bottom has logos for #MDAConference and Rare Disease Advisor.

#MDAConference: Jamie Moore, MD, Medical Director of the Pediatric Obesity Program at @childrenscolo.bsky.social, speaking about diet and neuromuscular disease at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #NeuromuscularDisease #Obesity

Quote from Abby Bronson, Vice-President of Patient Advocacy at Edgewise Therapeutics, discussing past challenges in Becker muscular dystrophy. Includes Rare Disease Advisor and MDA Conference logos.

#MDAConference: Abby Bronson, VP of Patient Advocacy at Edgewise Therapeutics, speaking Mar. 9 at the @mda.org's 2026 Clinical & Scientific Conference about efforts to reach out to patients with #Becker as a disease distinct from #Duchenne.

#RareDisease #MuscularDystrophy

A quote card from Rare Disease Advisor features Christopher Lewis at a conference. The quote talks about advancements in treating SMA and DMD since the 1950s, crediting his father's work. It includes a photo of Christopher Lewis holding a book. Logos for Rare Disease Advisor and MDA Conference are visible.

#MDAConference: Christopher Lewis, son of the late entertainer Jerry Lewis, who would have turned 100 on March 16, 2026. The younger Lewis was at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy

🧬 Two phase 3 trials are underway to evaluate salanersen in presymptomatic newborns with spinal muscular atrophy.

The STELLAR-1 and STELLAR-2 studies will assess outcomes with salanersen alone or following gene therapy.

Presented at the 2026 #MDAconference
#SMA #Neurology #GeneTherapy #RareDisease

Catch up with Hugh Bartlett at the #MDAconference from March 8-11 in Orlando, FL! Don’t miss the opportunity to explore the latest research advancements and clinical progress in #neuromuscular diseases.
To connect with Hugh: hbartlett@rarelifesolutions.com
#MuscularDystrophy #MDA #RareLifeSolutions

Today at the Muscular Dystrophy Association Clinical & Scientific Conference, Domi Stickens, PhD, CSO, presents a poster highlighting our RNA-targeted small-molecule therapeutics and first oral therapy in development for myotonic dystrophy type 1 (DM1).
#MDAconference #ArrakeensInAction