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

The image features a quote about Duchenne disease management from a healthcare professional associated with Children's Hospital Colorado. The background includes the logo for Rare Disease Advisor.

#MDAConference: Susan Apkon, MD, pediatric neurologist at @childrenscolo.bsky.social speaking March 9 about Duchenne muscular dystrophy at the 2026 @mda.org Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #DuchenneMuscularDystrophy

Targeting Skeletal Muscle in #DuchenneMuscularDystrophy: Integrating in Silico and Experimental Approaches to Sodium-Glucose Cotransporter-2 Inhibition. @ualberta.bsky.social

#OpenAccess text: https://loom.ly/M2rbNQk

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

Impaired stem cell migration and divisions in Duchenne muscular dystrophy revealed by live imaging - Nature Communications Using live-imaging in vivo and ex vivo, Sarde et al. show that dystrophic muscle stem cells have precocious differentiation through symmetric divisions and impaired migration, with differential impact...

My first-author PhD paper is out in @natcomms.nature.com 🙌 We developed unique in vivo/ex vivo live-imaging pipelines to uncover new muscle stem cell behaviors in #DuchenneMuscularDystrophy.
Big thanks to all co-authors & the @tajbakhshlab.bsky.social lab!
www.nature.com/articles/s41...

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/...

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....

DNA Damage Response Gene Upregulation May Predict Disease Severity in DMD In DMD, expression of the DNA damage response genes ETAA1, TOPBP1, and ATR correlates with severity, pointing to their potential as a biomarker panel.

Findings published in Pediatric Research suggest that the DNA damage response genes #ETAA1, #TOPBP1, and #ATR are upregulated in Duchenne muscular dystrophy (#DMD).

Learn more: https://bit.ly/4ks8UnE

#RareDisease #DuchenneMuscularDystrophy #MedSky

FDA Puts Cumberland’s Ifetroban on Fast Track for Cardiomyopathy in DMD The US FDA granted Fast Track designation to ifetroban, an oral thromboxane receptor antagonist, for cardiomyopathy in patients with DMD.

Cumberland Pharmaceuticals announced that the @fda.gov has granted #FastTrack Designation to #Ifetroban (#Dyscorban®), the company’s novel oral therapy for #Cardiomyopathy in patients with #DuchenneMuscularDystrophy (DMD).

Read more: https://bit.ly/4rTzo4j

#RareDisease #DMD #MedSky

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

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

“Transition” means more than healthcare. Patients with #DuchenneMuscularDystrophy and their families call for guidance, collaboration, and support across every area of life.

Read this topical review from the Special Issue on, “Survivorship in #ChildNeurology”

Read more - buff.ly/cxz1Rfe

Novel Therapeutics To Prevent And Treat Breast Cancer - Dr. Steven Quay MD, PhD, - CEO, Atossa Therapeutics, Inc. #BreastCancer #Zendoxifen #MetastaticDisease #MammographicBreastDensity #GlioblastomaMultiforme #DuchenneMuscularDystrophy www.youtube.com/watch?v=YThx...

Three-Year Results Show Greater Slowing of Progression With Elevidys® for Ambulatory DMD Sarepta announced the results of year 3 of the EMBARK trial, showing sustained functional benefits of delandistrogene moxeparvovec in DMD.

@sarepta.com announced positive 3-year functional outcomes from the #EMBARK study evaluating delandistrogene moxeparvovec-rokl in ambulatory patients with Duchenne muscular dystrophy (#DMD).

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

#RareDisease #DuchenneMuscularDystrophy #MedSky

#ArticleinPress: Targeting Skeletal Muscle in #DuchenneMuscularDystrophy: Integrating in Silico and Experimental Approaches to Sodium-Glucose Cotransporter-2 Inhibition. @ualberta.bsky.social

#OpenAccess text: https://loom.ly/M2rbNQk

Environmental Barriers Reduce Participation in Daily Life in Children With DMD Children with DMD participate less in daily life than their peers, largely due to a lack of adaptive modifications at home or at school.

Children with Duchenne muscular dystrophy (#DMD) participate less in daily life than do their typically developing peers, largely as a result of their physical environment. Study in Pediatric Neurology.

Read more: https://bit.ly/45Xmt8C

#RareDisease #DuchenneMuscularDystrophy

🚨 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

DMD Caregiving Negatively Affects Productivity, Pointing to Need for Better Support A survey of US-based DMD caregivers revealed the notable effects of caregiving demands on work and productivity.

Providing care for patients with Duchenne muscular dystrophy (#DMD) may significantly disrupt caregivers’ employment and productivity, according to a report published in Advances in Therapy

Read more: https://bit.ly/4qIKDM4

#RareDisease #DuchenneMuscularDystrophy

Dr. B.S. Rajput's Ortho Centre | Delhi & Mumbai Dr. B.S. Rajput, a leading Orthopedic Surgeon in Delhi & Mumbai, offers advanced treatments like Cellular Therapy, PRP & Tissue Regeneration for Knee, Hip & Joint Pain. Minimally Invasive Surgery for ...

Contact Us:
📞 +91-9820850187, +91-9820800187
🌐 drrajputorthocentre.com

Tags:
Duchenne muscular dystrophy treatment, long term case, bone marrow cell therapy, regenerative medicine, muscular dystrophy India, heart protection, muscle strength

#DuchenneMuscularDystrophy #CellularTherapy #DrBSRajput

🔍 TAGS:
duchenne muscular dystrophy, dmd diagnosis, dmd tests, blood test, emg ncv test, dystrophin gene analysis, muscular dystrophy in children, neuromuscular disease india, best dmd doctor india

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#dmdindia #duchennemusculardystrophy #musculardystrophyawareness #drbsrajput #earlydiagnosis

Dr. B.S. Rajput's Ortho Centre | Delhi & Mumbai Dr. B.S. Rajput, a leading Orthopedic Surgeon in Delhi & Mumbai, offers advanced treatments like Cellular Therapy, PRP & Tissue Regeneration for Knee, Hip & Joint Pain. Minimally Invasive Surgery for ...

👉 Early detection + right medical guidance बच्चों की quality of life को बेहतर बना सकती है।

अगर इनमें से कोई भी लक्षण दिखे, तो देरी न करें — समय रहते विशेषज्ञ से सलाह लें।
📞 Delhi / Mumbai: +91-9820850187, +91-9820800187
🌐 drrajputorthocentre.com

#DuchenneMuscularDystrophy #DMDSymptoms #DrBSRajput

Dr. B.S. Rajput's Ortho Centre | Delhi & Mumbai Dr. B.S. Rajput, a leading Orthopedic Surgeon in Delhi & Mumbai, offers advanced treatments like Cellular Therapy, PRP & Tissue Regeneration for Knee, Hip & Joint Pain. Minimally Invasive Surgery for ...

15+ वर्षों के clinical outcomes दिखाते हैं कि सही दिशा में इलाज से
complications कम और जीवन बेहतर हो सकता है।

📞 Delhi / Mumbai: +91-9820850187, +91-9820800187
🌐 drrajputorthocentre.com

#DuchenneMuscularDystrophy #DMDTreatment #DrBSRajput

In July's #EditorsChoice, Kamila Karimjee, Richard J. Piercy & co develop long-term, age-associated activity quantification for dog model of #DuchenneMuscularDystrophy #DMD

⚡ doi.org/10.1242/dmm....
📰 doi.org/10.1242/dmm....

💙 A Grandmother’s Perspective

Sarka Palouckova shares her experience caring for her 6-year-old grandson, William—and the strength, humor, and love that carry their family through life with #DuchenneMuscularDystrophy.

📖 Read Sarka’s story: https://bit.ly/4pNGiY2

#RareDisease #DMD

Obestatin treatment links mitochondrial homeostasis and skeletal muscle repair in Duchenne muscle dystrophy - Molecular Biomedicine Duchenne muscular dystrophy (DMD) is a genetic, progressive neuromuscular disease caused by mutations in the dystrophin protein which compromise the integrity of the sarcolemma. Current care of DMD in...

#Research
#Obestatin treatment links mitochondrial homeostasis and skeletal muscle repair in Duchenne muscle dystrophy doi.org/10.1186/s435...

#DuchenneMuscularDystrophy #Mitochondria #Utrophin #TFEB #NFATc1

Novel Medicines For Patients With Rare Diseases - Rich Daly - President and CEO, #CatalystPharmaceuticals Inc., joins me on Progress, Potential, And Possibilities #LambertEatonMyasthenicSyndrome #DuchenneMuscularDystrophy #Epilepsy #RareDiseases #OrphanDiseases www.youtube.com/watch?v=UrBV...

Histological comparison of muscle tissues from hDMD/mdx (control) and hDMDΔ52/mdx (humanized model of Duchenne muscular dystrophy) mice at 4, 16, 20, and 52 weeks. Panel A shows Masson’s trichrome-stained diaphragm sections, highlighting increased blue-stained fibrosis in hDMDΔ52/mdx over time. Panel B quantifies diaphragm fibrosis, with hDMDΔ52/mdx exhibiting significantly higher percentages at all ages (****P<0.0001). Panel C displays H&E-stained tibialis anterior muscle, revealing structural differences and more central nuclei in hDMDΔ52/mdx. Panel D quantifies central nuclei, showing marked elevation in hDMDΔ52/mdx across ages. Panel E plots fiber diameter distributions for both genotypes, indicating altered muscle morphology in hDMDΔ52/mdx.

In a new paper from Braunreiter, Kabadi & co @sarepta.com humanized mouse model of #DuchenneMuscularDystrophy displays molecular & functional deficits of #DMD that were restored by human-targeted therapy confirming its suitability for developing new treatments
journals.biologists.com/dmm/article/...

Deadline approaching! Apply for DDC’s Director of Research and Education role by Monday, 3rd November 2025.

Learn More: tinyurl.com/DDC-Research...

#NMD4C #DuchenneMuscularDystrophy #DMD #neuromusculardisease #NMD #RareDisease #ResearchLeadership #EducationStrategy #SeniorLeadership

Are you passionate about advancing research, advocacy, and education for people affected by #DuchenneMuscularDystrophy?
Defeat Duchenne Canada, one of NMD4C’s valued collaborators, is hiring a Director of #Research and #Education to drive their strategy

Learn More ➤ tinyurl.com/DDC-Research...

A timely and significant publication on gene therapy considerations for Duchenne muscular dystrophy. @worldduchenne.bsky.social @duchennenl.bsky.social #Duchennemusculardystrophy #DMDwww.sciencedirect.com/science/article/pii/S096...

Combination PMO Treatment Holds Promise for DMD Researchers developed a TGF-β-targeting PMO that could be beneficial in DMD when used in combination with dystrophin exon skipping PMO.

A team of researchers from the US and Korea developed a TGF-β-#TPMO that could be beneficial in Duchenne muscular dystrophy (#DMD) when used in combination with #DPMO. @moltherapy.bsky.social

Read more: https://bit.ly/4gV0LX8

#RareDisease #DuchenneMuscularDystrophy #MedSky