📄 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

DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene that leads to the loss of the dystrophin protein. Exon-skipping therapy with phosphorodiamidate morpholino oligomers (PMOs) has been shown to restore the disrupted...

Thrilled to share our new Methods in Molecular Biology chapter. link.springer.com/protocol/10....　We describe in vivo methods for systemic DG9-PMO delivery, exon 51 skipping, and efficacy assessment in hDMDdel52;mdx mice. #DMD #RNAtherapeutics #ASO #DG9 #ExonSkipping @SpringerNature

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈

Support World Duchenne Awareness Day 2025 with DMDWarrior

Theme of 2025: Make Cures Affordable for Duchenne

#WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

Support World Duchenne Awareness Day 2025 with DMDWarrior World Duchenne Awareness Day will be observed globally on September 7, 2025, with the theme “Make Cures Affordable for Duchenne.” This important day raises awareness about Duchenne Muscular Dystrophy (DMD), a rare genetic condition that causes progressive muscle weakness and affects thousands of individuals and families worldwide. Each year, the campaign calls for more attention, research, and action toward better treatments and, ultimately, a cure. Theme of 2025: Make Cures Affordable for Duchenne This year’s theme reflects the urgent and universal wish of the Duchenne community: access to effective and affordable cures for Duchenne Muscular Dystrophy (DMD). Families, patients, and advocacy groups are united in calling for equitable access to treatments, accelerated research, and global collaboration.

🎈 SEPTEMBER 7, 2025 🎈

Support World Duchenne Awareness Day 2025 with DMDWarrior

Theme of 2025: Make Cures Affordable for Duchenne

#WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD
#UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

Exon Skipping and Inclusion Therapies This book presents a collection of protocols reflecting the latest advancements in exon skipping and inclusion strategies.

🎉 New Release! The 2nd Edition of Exon Skipping and Inclusion Therapies is now published with Springer Nature!
This edition highlights new breakthroughs in RNA-targeted therapies.
Grab your copy
link.springer.com/book/10.1007...
#ExonSkipping #RNAtherapeutics #GeneTherapy #DMD #SMA #SpringerNature

www.preprints.org/manuscript/2... Excited to share our latest preprint:
🧬 “Brogidirsen and Exon 44 Skipping for DMD: Advances and Challenges in RNA-Based Therapy”
📄 www.preprints.org/manuscript/2...
Proud of Annie Tang’s excellent work leading this review. #DMD #RNAtherapy #exonskipping #ASO

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy Dyne Therapeutics, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping in the Phase 1/2 DELIVER global clinical study.

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

#dmd #duchenne #dyne #dyne-251 #exon51 #exon51skipping #exonskipping #ema #orphandrug

Entrada Therapeutics Receives FDA Approval to Begin Clinical Trial of ENTR-601-44 Exon Skipping Treatment Entrada Therapeutics, announced that the United States Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping.

Entrada Therapeutics Receives FDA Approval to Begin Clinical Trial of ENTR-601-44 Exon Skipping Treatment

👉 Read More: dmdwarrior.com

#dmd #duchenne #exon44 #exon44skipping #exonskipping

FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04) Nippon Shinyaku declared that NS-051/NCNP-04, a medication being developed to treat Duchenne Muscular Dystrophy (DMD), has been granted Rare Pediatric Disease Designation by the Food and Drug Administration (FDA). Nippon Shinyaku and the National Center of Neurology and Psychiatry jointly discovered the antisense oligonucleotide NS-051/NCNP-04. By omitting a portion of the dystrophin gene’s genetic material, NS-051/NCNP-04 creates a functioning dystrophin protein with a little shorter chain length, which should prevent the decline in muscle function.

FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)

👉 Learn More: dmdwarrior.com

#dmd #duchenne #bmd #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #exon51 #exon51skipping #nipponshinyaku #exonskipping

Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne Nippon Shinyaku Corporation has announced the first human trial results of its drug 'Brogidirsen' (NS-089/NCNP-02), which was developed for exon 44 skipping in the treatment of Duchenne muscular dystrophy (DMD). The National Center of Neurology and Psychiatry is announced the publication of a research paper in Cell Reports Medicine detailing the results of an investigator-initiated trial for NS-089/NCNP-02, known as “brogidirsen.” This innovative treatment for Duchenne muscular dystrophy (DMD), jointly developed with Nippon Shinyaku Co., Ltd., represents a significant advancement in exon 44 skipping therapy. What is Brogidirsen? Brogidirsen (NS-089/NCNP-02) is a nucleic acid drug co-discovered by Nippon Shinyaku and NCNP, and is expected to be a therapeutic drug for DMD patients with dystrophin gene mutations amenable to exon 44 skipping.

Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne

What is Brogidirsen?

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #nipponshinyaku #brogidirsen #NS089 #exonskipping #exon44

Duchenne Muscular Dystrophy: Treatment & Cost - DMD Warrior Learn about Duchenne Muscular Dystrophy (DMD), its early symptoms, current cures and treatment cost, gene therapies and the future of patients in the World.

Duchenne Muscular Dystrophy: Treatment & Cost

👉 Read and Share: dmdwarrior.com/duchenne-mus...

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna #elevidys #exonskipping #exon