Impact of C4BPA on Muscle progenitor cell differentiation: insights for Duchenne muscular dystrophy treatment - Cell Death & Disease Cell Death & Disease - Impact of C4BPA on Muscle progenitor cell differentiation: insights for Duchenne muscular dystrophy treatment

Congratulations to the team for a new paper published 👏

🧫By combining advanced co-culture systems, secretome profiling, and 3D engineered muscle models, we investigated how #Duchenne muscular dystrophy (DMD)-derived FAPs inhibit myogenesis.

bit.ly/3PE1Hpv

Muscular Dystrophy (Duchenne & Becker)

Genetic disorder causing progressive muscle weakness due to dystrophin defect.
Early onset (Duchenne) = severe | Later onset (Becker) = milder.
#MuscularDystrophy #Duchenne #Becker #Neurology #VikaasShandily

#NMD4C #DMD #Duchenne #musculardystrophy

Regenxbio, Solid report data with Duchenne gene therapies Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

This week saw both #Regenxbio and #SolidBio report new #data with their #genetherapy candidates for #Duchenne muscular dystrophy (DMD), vying to join Sarepta's Elevidys on the market.

Proud to see this work from Dr. Pesco reviewing the expanding therapeutic landscape in DMD.
Important synthesis of dystrophin restoration and emerging disease-modifying strategies as the field rapidly evolves.

www.mdpi.com/2813-0413/5/...

#DMD #Duchenne #Neuromuscular #RareDisease

#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

REGENXBIO Unveils Promising Interim Data from Phase I/II Trial for Duchenne's Disease Treatment REGENXBIO announces encouraging interim results from its RGX-202 trial, showing promise for Duchenne muscular dystrophy treatment with improved functionality and stability.

REGENXBIO Unveils Promising Interim Data from Phase I/II Trial for Duchenne's Disease Treatment #United_States #Rockville #Duchenne #REGENXBIO #RGX-202

#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

A historical perspective on the development of antisense oligonucleotide treatments for Duchenne muscular dystrophy and spinal muscular atrophy - Annemieke Aartsma-Rus, Shin'ichi Takeda, 2026 Splice modulating antisense oligonucleotides (ASOs) have been approved for the treatment of spinal muscular atrophy (nusinersen) and Duchenne muscular dystrophy...

From the JND special issue on RNA therapeutics:
Aartsma-Rus & Takeda examine the development of ASO therapies for #Duchenne muscular dystrophy and #SMA.

The article highlights scientific, clinical and regulatory lessons for future therapies.

Read it here: buff.ly/rJJb5N7

#Neuromuscular #DMD #SMA

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

Celebrating the 2026 Advocacy Leadership Awards for Duchenne Research Leaders The 2026 Advocacy Leadership Awards recognize key bipartisan leaders in Congress advocating for Duchenne muscular dystrophy research and care advancements.

Celebrating the 2026 Advocacy Leadership Awards for Duchenne Research Leaders #United_States #Washington #Duchenne #PPMD #FED

PPMD Partners with CINRG to Enhance Duchenne Natural History Study PPMD has teamed up with the Cooperative International Neuromuscular Research Group to enrich the Duchenne Natural History Study, driving insights for better care.

PPMD Partners with CINRG to Enhance Duchenne Natural History Study #United_States #Washington #Duchenne #PPMD #CINRG

REGENXBIO Unveils Groundbreaking Findings at MDA 2026 Conference on Duchenne Therapy REGENXBIO Inc. highlights its pivotal research on RGX-202, a novel gene therapy for Duchenne muscular dystrophy at the upcoming MDA conference in Orlando.

REGENXBIO Unveils Groundbreaking Findings at MDA 2026 Conference on Duchenne Therapy #United_States #Orlando #Duchenne #REGENXBIO #RGX-202

Illustration by Guillaume-Benjamin Duchenne, from Mécanisme de la physionomie humaine (1862).

Source: National Science and Media Museum / Flickr: The Commons

pdimagearchive.org/images/b489328c-388a-4f1...

#electricity #duchenne #art #publicdomain

Episode 160: Early Pathology, Biomarkers, and the Next Phase of DMD Care | NeurologyLive - Clinical Neurology News and Neurology Expert Insights Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jeff Chamberlain, PhD. [LISTEN TIME: 23 minutes]

🎙️ New Mind Moments Ep.

During #DMD Awareness Week, Jeff Chamberlain (U. of Washington School of Medicine) discusses early DMD pathology, biomarkers, immunologic drivers, & what gene therapy may mean for 2026+.

🎧 Listen: www.neurologylive.com/view/episode...
#Duchenne #GeneTherapy #Neurology #Neuro

First Patient Dosed in Phase 2 Pediatric Trial Evaluating SAT-3247 for DMD The first participant has received treatment in the BASECAMP trial evaluating SAT-3247, an investigational AAK1 inhibitor for DMD.

@satellosbio.bsky.social announced that the first patient has been dosed in a phase 2 pediatric #ClinicalTrial evaluating SAT-3247, an investigational therapy for #Duchenne muscular dystrophy (#DMD).

Learn more: https://bit.ly/46cLHQH

#RareDisease #SAT3247 #MedSky

¡Ayer lanzamos Viviendo la Diversidad Podcast!
Las primeras opiniones recibidas son geniales y nos emocionan.
¡Os animamos a ver/escuchar el episodio! ❤

youtu.be/YQagBBFEVhk

🔁 Comparte para llegar a más personas.

#Podcast #Discapacidad #Duchenne #DiversidadFuncional #SexualidadYDiscapacidad

PTC pulls file for Duchenne therapy on FDA feedback PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

#PTCTherapeutics has abandoned its latest attempt to secure #FDA approval for #Duchenne muscular dystrophy (DMD) treatment #Translarna, after the FDA said it was unlikely to approve the drug based on the submitted data.

Join us and Muscular Dystrophy Canada at our upcoming CPD-accredited webinar, on #DMD in the New Steroid Age: Practical Considerations" taking place on April 7, 2026 at 5pm ET.

🔗 | Register here: neuromuscularnetwork.ca/event/17-02-...

#NMD4C #letstalkNMD #duchenne #steriod #neuromuscular

Screening for brain‐related comorbidities in #Duchennemusculardystrophy: Construction, reliability, and validity of the BIND screener
#Duchenne #neurodevelopment #neuropsychiatry

onlinelibrary.wiley.com/doi/10.1111/...

»A #Duchenne smile involves contraction of both the zygomatic major muscle (which raises the corners of the mouth) and the orbicularis oculi muscle (which raises the cheeks and forms crow's feet around the eyes). The (...) has been described as "smizing", as in "smiling with the eyes".« (Wiki)

New Treatment Option (SAT-3247) for Duchenne Muscular Dystrophy Shows Promise in Early Phase Trial Wildon Farwell, MD, CMO at Satellos, discusses the safety and efficacy of SAT-3247, a new treatment option for Duchenne muscular dystrophy.

In a recent @CheckRare feature, Satellos CMO Dr. Wildon Farwell discusses the science behind SAT-3247 and how restoring the #muscleregeneration process could offer a new therapeutic approach for people living with #Duchenne #FSHD $msclf $mscl Watch the interview checkrare.com/new-treatmen...

Newborn Screening for Duchenne: Clinical and Policy Implications | NeurologyLive - Clinical Neurology News and Neurology Expert Insights Paul Melmeyer, PhD, executive vice president of public policy and advocacy at the Muscular Dystrophy Association, explains how adding Duchenne muscular dystrophy to the Recommended Unified Screening P...

🧬 Newborn Screening for #Duchenne (DMD) is here❗
DMD & MLD were added to the US RUSP—unlocking earlier diagnosis, earlier treatment, and stronger family support 🍼💪

Read more: www.neurologylive.com/view/newborn...
#RareDisease #Neurology #NewbornScreening #Neuromuscular #PublicHealth #GeneticTesting

Best Short Documentary in Milan 🇮🇹 for #documentary ‘Standing Strong’ (Samen Sterk)!
An award I’m honoured to share with Leo, Lewis, Bart, Tom and their families, friends and volunteers whose resilience impressed me. And with #Duchenne Parent Project in The Netherlands for a wonderful collaboration.

🚨 Interesting article by @Weekademia lab from @UniNorthants

A unified model for Duchenne muscular dystrophy gene involvement in cancer: context-dependent tumour suppression and oncogenicity

👉 buff.ly/9KdJjJj

#RareDisease #DuchenneMuscularDystrophy #cancer #tumour
#DMD #duchenne

PPMD Celebrates a Victorious Weekend at the 2026 Disney Marathon in Orlando At the 2026 Walt Disney World® Marathon Weekend, PPMD achieved remarkable success by raising $300,000 and securing two race victories.

PPMD Celebrates a Victorious Weekend at the 2026 Disney Marathon in Orlando #United_States #Orlando #Duchenne #PPMD #Jack_Napoli

U.S. Health Department Includes Duchenne in Newborn Screening Panel for Early Detection The U.S. Department of Health and Human Services has included Duchenne muscular dystrophy in the newborn screening panel, enhancing early care for affected infants.

U.S. Health Department Includes Duchenne in Newborn Screening Panel for Early Detection #United_States #Washington #Duchenne #PPMD #MDA

Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. #DMD 👉 Read More: DMDWarrioR.com

Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy

👉 Read More: DMDWarrioR.com

Ractigen Therapeutics Begins Groundbreaking Study of saRNA Treatment for Duchenne Muscular Dystrophy Ractigen Therapeutics announces the first patient has been dosed in a clinical trial for RAG-18, a novel saRNA treatment for Duchenne Muscular Dystrophy, offering new hope.

Ractigen Therapeutics Begins Groundbreaking Study of saRNA Treatment for Duchenne Muscular Dystrophy #China #Beijing #Duchenne #Ractigen #RAG-18