Latest posts tagged with #dmd on Bluesky
🚀 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
#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
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
#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
#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
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
🧬 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
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.
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...
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
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 #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/...
🎙️ 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....
@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
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
Congrats to Chloe Li on her successful PhD oration on muscle plasticity in DMD. Great job Chloe! Thesis to follow soon
Proud supervisors: @kristyswid.bsky.social, @justinhardeephd.bsky.social & Gordon Lynch
#muscle #DMD @unimelb.edu.au @mda.org @afm-telethon.bsky.social @worldduchenne.bsky.social
Éxito I #cineforum de #dmd #huesca con aforo completo
derechoamorir.org/2026/02/16/celebrado-i-c...
#eutanasia #muertedigna #testamentovital #derechoamorir #derechosfinaldevida #aragon #asociaciondmd
Why build DnD wizards to be durable? Because I want tos survive to continue playing a wizard!
#dmd #characteroptimization #wizard
