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DMD Warrior
@dmdwarrior.com
43 followers 10 following 110 posts
Stay informed on #DMD cures, clinical trials, and #Duchenne muscular dystrophy research. Visit DMDWarrior.com for updates and support.
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DMD Warrior @dmdwarrior.com · 7d
REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program

#dmd #duchenne #regenxbio #genetherapy #clinicaltrials #rgx202

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REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program REGENXBIO announced it has completed enrollment of 30 participants in the pivotal portion of its AFFINITY DUCHENNE trial for RGX-202, an investigational gene therapy for Duchenne muscular dystrophy.
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DMD Warrior @dmdwarrior.com · 7d
Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy

#dmd #duchenne #imlifidase #genetherapy #clinicaltrials

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Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy Two gene therapy trials, one sponsored by Sarepta and the other sponsored by Genethon, support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapy.
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DMD Warrior @dmdwarrior.com · 15d
Why Elevidys Was Not Approved by the European Medicines Agency (EMA)?

You can access the comprehensive report on the reasons for the Elevidys refusal published by EMA from our web page link.

#dmd #duchenne #elevidys #eu

👉 Read More: DMDWarrioR.com
If you're searching for answers to 'why Elevidys was not approved by the European Medicines Agency (EMA)', you're not alone. In this article, we explore the key reasons behind the EMA’s decision and what it means for patients, families, and the future of gene therapy in the EU.
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DMD Warrior @dmdwarrior.com · 23d
Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping

#dmd #duchenne #exon44 #brogidirsen

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Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089/NCNP-02). #dmd #duchenne #exon44 #brogidirsen 👉 Read More: DMDWarrioR.com
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DMD Warrior @dmdwarrior.com · 24d
Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026

#dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences

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Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026
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DMD Warrior @dmdwarrior.com · 24d
Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production?

#pemf #dmd #duchenne

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Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production? In countries like Uzbekistan, information is being shared about the potential treatment of Duchenne Muscular Dystrophy with PEMF therapy. Pulsed Electromagnetic Field (PEMF) therapy is not a cure for Duchenne Muscular Dystrophy. Be careful not to let scammers take your money.
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DMD Warrior @dmdwarrior.com · Sep 30
Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy

#dmd #duchenne
Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv).
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DMD Warrior @dmdwarrior.com · Sep 28
Cures of #Duchenne (Detailed List of All Clinical Trials for #DMD)

List of all researches such as #gene therapy, exon skipping, reducing inflammation and improving #muscle growth & protection cures for duchenne #muscular dystrophy.

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Cures of Duchenne (Detailed List of All Clinical Trials) List of all researches such as gene therapy, exon skipping, reducing inflammation and improving muscle growth & protection cures for duchenne muscular dystrophy.
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DMD Warrior @dmdwarrior.com · Sep 10
Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial

#dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences

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Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial With del-zota, normal dystrophin increased by 25% and creatine kinase decreased by 80%. Among exon 44 skipping therapies, the highest values ​​currently received have caused excitement. Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting sustained muscle fiber protection.
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DMD Warrior @dmdwarrior.com · Sep 9
Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy

#dmd #duchenne #BBMD101 #BeliefBioMed #clinicaltrials

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Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy Belief BioMed, an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the registration clinical trial (CTR20252461) of its independently developed and produced BBM-D101 injection for Duchenne Muscular Dystrophy (DMD) has successfully completed the first patient dosing in Beijing, China.
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DMD Warrior @dmdwarrior.com · Sep 9
Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males

#dmd #duchenne #sgt003 #genetherapy #clinicaltrials

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Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males The phase 3 study (NCT07160634) of the SGT-003 gene therapy developed by Solid Biosciences is expected to begin in October 2025 and be completed in January 2029. The SGT-003 Phase 3 study will include 80 ambulant males, moreover the study locations have not yet been shared.
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DMD Warrior @dmdwarrior.com · Sep 8
Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting

#dmd #duchenne #ker065

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Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting Phase 1 Trial in Healthy Participants of KER-065, a Modified Activin Receptor Ligand Trap, Supports Development in Duchenne Muscular Dystrophy and Bone Disorders.
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DMD Warrior @dmdwarrior.com · Sep 7
GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium

Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD).

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GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase 1/2 study aims to evaluate the tolerability, safety, and efficacy of the drug GNR-097. #dmd #duchenne #gnr097 #genetherapy #clinicaltrials 👉 Read More: DMDWarrioR.com
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DMD Warrior @dmdwarrior.com · Sep 6
🎈 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
🎈 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
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DMD Warrior @dmdwarrior.com · Sep 6
How Does My Child Participate in Clinical Trials for Duchenne?

Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you.

Learn More 👇

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How Does My Child Participate in Clinical Trials for Duchenne? Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you. If your child has been diagnosed with Duchenne muscular dystrophy (DMD), you may be exploring every possible option for treatment and care—including clinical trials. But how does your child participate in a Duchenne clinical trial? And where can you find trials that are actively recruiting?
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DMD Warrior @dmdwarrior.com · Aug 30
🎈 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.
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DMD Warrior @dmdwarrior.com · Aug 25
ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy

#givinostat #duvyzat

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ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy Positive long-term safety and efficacy data for Duvyzat (Givinostat) as a treatment for Duchenne muscular dystrophy (DMD) were published today by ITF Therapeutics, an Italfarmaco affiliate. The data came from the company's open-label extensions of its Phase 2 and Phase 3 (EPIDYS) trials. 🎈 New data published in Annals of Clinical and Translational Neurology show that long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with Duchenne muscular dystrophy. 🎈 Meaningful clinical benefit was observed across treatment groups, regardless of disease stage at initiation. 🎈 Givinostat remained well-tolerated during extended use, consistent with previous clinical trials. 🎈 Average follow-up in the extension study exceeded 36 treatment months, with some patients receiving treatment for over eight years since initiating givinostat treatment. #givinostat #duvyzat 👉 Read More: DMDWarrioR.com
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DMD Warrior @dmdwarrior.com · Aug 19
Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026

#agamree #vamorolone #uae #bahrain #oman #saudi_arabia #kuwait

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Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026 Santhera Pharmaceuticals announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE (vamorolone) in the United Arab Emirates, the Kingdom of Saudi Arabia, Kuwait, Oman and Bahrain, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.
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DMD Warrior @dmdwarrior.com · Jul 29
FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’

#dmd #sarepta #roche #elevidys #fda #duchenne

👉 Read More: DMDWarrioR.com
FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’ In a statement Monday, it said the FDA's swift action eliminated a problem with Sarepta, allowing Elevidys to return to the market without another safety study as had been feared.
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DMD Warrior @dmdwarrior.com · Jul 26
FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys

#dmd #sarepta #roche #elevidys #fda #duchenne

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FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns. The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.
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DMD Warrior @dmdwarrior.com · Jul 25
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

#dmd #sarepta #roche #elevidys #europe #ema

👉 Read More: DMDWarrioR.com
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities. Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).
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DMD Warrior @dmdwarrior.com · Jul 23
Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

#dmd #sarepta #roche #elevidys

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Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States
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DMD Warrior @dmdwarrior.com · Jun 15
Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients.

#dmd #sarepta #roche #elevidys

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Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients. Sarepta reports that Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place.
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DMD Warrior @dmdwarrior.com · May 28
Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy

#dmd #duchenne

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Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy Entrada Therapeutics Receives Authorization in the European Union to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-45 in Patients Living with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping. Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR) to initiate ELEVATE-45-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-45 in patients living with Duchenne muscular dystrophy (DMD) who are amenable to exon 45 skipping.
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DMD Warrior @dmdwarrior.com · May 5
Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy

👉 DMDWarrioR.com

#dmd #duchenne #deramiocel #cardiomiopathy
Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy Capricor Therapeutics , a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company’s Biologics License Application (BLA) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. Is Deramiocel FDA Approved? During the meeting, FDA stated that no significant deficiencies have been identified by the Review Committee and that the package is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. The FDA has also confirmed its intent to hold an advisory committee meeting, although an official date has not yet been set.
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