Abrazando a Noelia en la distancia.

Buen viaje, Noelia #derechoamorir #DMD

#NMD4C #DMD #Duchenne #musculardystrophy

🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TRdbxW #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT

📄 From our special issue on RNA therapeutics:
A study examining cardiac outcomes in DMD treated with the exon-skipping therapy eteplirsen.

Treated patients showed slower LVEF decline and lower risk of reaching cardiac dysfunction thresholds. buff.ly/ycbsqVl

#DMD #RNATherapeutics #ExonSkipping

🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TRSr0P #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT

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: 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 🎥

🧬 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

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

Pooled Analysis Suggests Manageable Safety for Delandistrogene Moxeparvovec in DMD A pooled analysis suggests manageable safety for delandistrogene moxeparvovec in DMD, with most adverse events occurring early.

#MDAConference: A pooled safety analysis based on data from phase 1 through phase 3 #ClinicalTrials of delandistrogene moxeparvovec suggests a manageable safety profile in ambulatory patients with Duchenne muscular dystrophy (#DMD). @mda.org

Learn more: https://bit.ly/4bcBmWv

Our recent podcast episodes cover building an optimized DnD Pugilist. We picked species, and our choices here were more impactful than we anticipated.
#dmd #pugilist

Z-Rostudirsen for DMD May Improve Muscle Function, With Signals of Cardiopulmonary Benefit Data from the DELIVER trial of z-rostudirsen in DMD showed sustained improvement in muscle function and potential cardiopulmonary benefit.

#DYNE251, an investigational exon 51-skipping therapy, provides sustained improvements in muscle function and potentially preserves #Cardiopulmonary function in patients with #DMD. Data to be presented at the 2026 @mda.org #MDAConference

Learn more: https://bit.ly/46C0ZyI

#RareDisease #MedSky

Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy

Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera

SPARC Portal The open community platform for bridging the body and the brain through neuroscience and systems physiology data, computational and spatial modeling, and device design.

Don't Miss: The Design of Medical Devices Conference (April 21–22) marks 25 years of cross-sector collaboration in medical technology. Keynote by Dr. Tyler Best of ARPA-H.

Explore open data for device research at https://sparc.science

Register: sparc.science/news-a...

🧪 #MedicalDevices #DMD

🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TRHLBh #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT

DMD kas hastası olan kardeşler için zamana karşı yarış - İlkses Gazetesi Diyarbakır’ın Çınar ilçesinde yaşayan Esen Gülmüş, DMD kas hastası ve kalp sorunları bulunan 3,5 yaşındaki Adem ile 1,5 yaşındaki Yusuf için yardım çağrısında b

DMD kas hastası olan kardeşler için zamana karşı yarış #DMD #hastalık #destek #yardım

🧬 New Special Issue of JND out today to celebrate #RareDiseaseDay:
“RNA Therapeutics in Neuromuscular Disorders”

Exploring RNA-targeted approaches in Duchenne muscular dystrophy, Spinal muscular atrophy, Myotonic dystrophy and more: buff.ly/JOtdEJb
#Neuromuscular #RNAtherapeutics #DMD #SMA #DM1

Scan this QR code to register for access to our Dossier on Duchenne muscular dystrophy, #DMD.

On #RareDiseaseDay, we are offering researchers access to a Dossier on Duchenne muscular dystrophy, #DMD, a rare, progressive neuromuscular disorder caused by mutations in the dystrophin gene.

Intercellular communication from myotubes to fibroblasts is differentially impacted by mineralocorticoid receptor signaling and muscular dystrophy - Cell Communication and Signaling Cell Communication and Signaling - Replacement of striated muscle with fibrosis leads to the symptoms and fatality in Duchenne muscular dystrophy (DMD). Transient local production of the...

Happy to share our recent publication on intercellular communication from the Rafael-Fortney lab. Check this one out!
#fibroblasts #myotubes #DMD #skeletalmuscle #fibrosis

link.springer.com/article/10.1...

New Noninvasive Indices Identify Early Myocardial Dysfunction in DMD-Associated Cardiomyopathy Three newly developed noninvasive indices can accurately detect early myocardial dysfunction in patients with DMD-associated cardiomyopathy.

Newly developed noninvasive indices can accurately detect early myocardial dysfunction in patients with Duchenne muscular dystrophy (#DMD)-associated #Cardiomyopathy. Study in Pediatric Cardiology.

Learn more: https://bit.ly/4r0q6m3

#RareDisease #DuchenneMuscularDystrophy #MedSky

Final CIFFREO Trial Results: No Benefit, Poor Safety for Fordadistrogene Movaparvovec in DMD Final results from the phase 3 CIFFREO trial of fordadistrogene movaparvovec demonstrated no improvement in ambulatory boys with DMD.

According to final findings from the phase 3 #CIFFREO trial, a single intravenous dose of fordadistrogene movaparvovec does not improve functional outcomes in ambulatory boys with Duchenne muscular dystrophy (#DMD). Reported in @thelancetneuro.bsky.social

Read more: https://bit.ly/4rE4GfL

Mechanism-guided Untargeted-to-targeted Lipidomics Identifies Phosphatidylcholine 38:4 in Rat Bile as an Abcb4/Mdr2 Inhibition Marker Drug-induced liver injury (DILI) can occur when the canalicular phospholipid floppase MDR3 (Mdr2 in rodents) is inhibited, but there is still a lack of early biomarkers to detect this risk. In this st...

Mechanism-guided Untargeted-to-targeted Lipidomics Identifies Phosphatidylcholine 38:4 in Rat Bile as an Abcb4/Mdr2 Inhibition Marker #DMD #MassSpec dmd.aspetjournals.org/article/S009...

A double-blind study documents the complex link between corticosteroid-related height stunting, weight gain, and motor function in #DuchenneMuscularDystrophy #DMD

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

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

Figure showing progressive cardiac abnormalities in DmdΔ45 rats compared with wild‑type controls. Panel A presents a bar graph of heart weight normalized to body weight, with DmdΔ45 rats showing increased values. Panel B displays heart tissue sections: HPS staining on the left reveals structural disorganization, and Sirius Red staining on the right highlights increased fibrosis in DmdΔ45 hearts, particularly in the left and right ventricles. Panel C shows a line graph of cardiac output measured by transthoracic echocardiography from 3 to 18 months, with DmdΔ45 rats exhibiting a decline over time. Panel D presents left ventricular end‑systolic diameter measurements, which are enlarged in DmdΔ45 rats. Panel E shows a bar plot of ejection fraction, reduced in DmdΔ45 animals.

Tao Wang, Cynthia Daoud, Abbass Jaber, Isabelle Richard & co generated a new #RatModel of #DuchenneMuscularDystrophy with an exon 45 deletion using CRISPR/Cas9, recapitulating the most-frequent single-exon deletion and key features of #DMD.

Read: doi.org/10.1242/dmm....

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

FDA Clears First-in-Class PBGENE-DMD to Begin Phase 1/2 Trial in Duchenne Muscular Dystrophy Precision BioSciences has received FDA clearance to launch a phase 1/2 trial of PBGENE-DMD in ambulatory DMD.

PBGENE-DMD, Precision BioSciences’ investigational in vivo gene editing therapy for ambulatory patients with Duchenne muscular dystrophy (#DMD), has cleared a key regulatory milestone.

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

#PBGENEDMD #GeneTherapy #MedTwitter