Duchenne Muscular Dystrophy (DMD) is a genetic disorder that causes progressive muscle weakness.
There’s no cure yet, but research is bringing hope closer every day.
Learn. Share. Support. 💙
#DuchenneAwareness #EndDuchenne
Did you know that I carry Duchenne Muscular Dystrophy and have two sons with this terminal genetic illness?
9/7 is World Duchenne Awareness Day!
#CureDuchenne #WorldDuchenneAwarenessDay #duchenneawareness #duchennemusculardystrophy #duchenne
Assemblymember Boerner takes a picture with Nicholas Archdeacon in her Capitol Office.
📢 #AB242 Passes Assembly Health with Unanimous & Bipartisan Support! 💙
AB 242 would add DMD to CA's Newborn Screening Program, ensuring early diagnosis & timely treatment, giving families hope.
Thank you to Leeandra & Nicholas Archdeacon for your tireless advocacy! 💙👏
#DuchenneAwareness
Join Henry's mission to support Duchenne UK! How can we all contribute to vital research for Duchenne Muscular Dystrophy? #DuchenneAwareness
www.justgiving.com/campaign/henrysmission
The Challenge of Getting DMD Gene Therapy: Is Geography Destiny? Over the last decade, significant advancements have been made in the development of gene therapies for DMD, offering hope for a life-changing treatment. However, despite the promise these therapies hold, a major obstacle stands in the way of many patients: the prohibitively high cost of these treatments. This article explores the reasons behind the exorbitant price of Duchenne gene therapies and the devastating consequences for patients, families, and the healthcare system as a whole.
The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?
#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness
👉 Read More: dmdwarrior.com
Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers SAT-3247 Announcement Summary 📌Company remains on track to report Phase 1a data from both the Single- and Multiple-Ascending Dose (SAD and MAD) cohorts in the healthy volunteer portion of the study in 1Q 2025 at an upcoming major medical meeting 📌The Phase 1b portion of the trial, in DMD patients, is underway with the intention of enrolling up to 10 adult volunteers with genetically confirmed DMD during 1Q 2025 📌Phase 2 IND filing on track to be submitted by end of 1Q 2025
Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers
#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #satellos
👉 Read More: dmdwarrior.com
Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping) Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping Treatment), a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-44 in Duchenne Muscular Dystrophy Patients. ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for the Clinical Trial of an Investigational Medicinal Product. The study aims to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 44 skipping.
Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping)
👉 Read More: dmdwarrior.com
#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #uk #england #exon44skipping #entrada
dmd warrior dmdwarrior
WE WILL FIGHT DUCHENNE and WE WILL WIN 💪
👉 dmdwarrior.com
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna
Before we knew that DMD even existed we thought that what we now know to be #pseudohypertrophy was just awesome healthy calf muscles. Sadly, we know better.
#duchennemusculardystrophy #dmd #duchenneawareness
youtu.be/vQMbzexFb5Q?...
Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy Solid Biosciences shared detailed information about the clinical trials of its SGT-003 Gene Therapy developed for the treatment of Duchenne Muscular Dystrophy. Solid Biosciences shared that four patients have been dosed in the SGT-003 gene therapy clinical trial; it was well tolerated in all patients with no side effects observed; and data from the first three patients could be available in Q1 2025.
Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy
👉 Read More: dmdwarrior.com
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneawareness #gene #dna #solidbiosciences #sgt003
Vamorolone (Agamree) Approved in Scotland The Scottish Medicines Consortium (SMC) approved the Agamree (Vamorolone) for use by NHS Scotland. The medication vamorolone, also marketed under the brand name Agamree, has been approved by the Scottish Medicines Consortium (SMC) as a therapy option for Duchenne muscular dystrophy in patients four years of age and older in Scotland as of today, Monday, January 13 2025.
Vamorolone (Agamree) Approved in Scotland
👉 Read More: dmdwarrior.com
#dmd #duchenne #bmd #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna #agamree #vamorolone #santhera #scotland #uk #england
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
Is Duchenne Gene Therapy a Suitable Treatment Despite its Immunogenic Class Effect? This article will examine current gene therapies up to August 2024, including micro-dystrophin Duchenne therapies and CRISPR-Cas9 therapies found on ClinicalTrials.gov. Progressive muscle weakness and eventual mortality as a result of cardiomyopathy or respiratory complications are the hallmarks of Duchenne muscular dystrophy (DMD), a severe X-linked disorder. At present, there is no cure for DMD, and the primary focus of standard treatments is on symptom management. Gene therapies can more effectively address the genetic cause of the disease by employing immunosuppressive measures and optimized vector designs. What are the effects of gene therapy treatments in Duchenne muscular dystrophy (DMD) disease, do gene therapies work? Here is a comprehensive analysis.
Is Duchenne Gene Therapy a Suitable Treatment Despite its Immunogenic Class Effect?
Read More: dmdwarrior.com/is-duchenne-...
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna
Zebrafish Modeling Shows Promise in Duchenne (DMD) Research Zebrafish (Danio rerio) has emerged as a valuable model organism for investigating musculoskeletal development and the pathophysiology of associated diseases. Key genes and biological processes in zebrafish are very similar to those in humans, and the rapid development and transparent embryos make zebrafish ideal for in vivo studies of bone and muscle formation as well as the molecular mechanisms underlying musculoskeletal disorders. Research is focusing on the utility of zebrafish in modeling various musculoskeletal disorders, with particular emphasis on bone diseases such as osteoporosis and osteogenesis imperfecta, and muscle disorders such as Duchenne muscular dystrophy.
Zebrafish Modeling Shows Promise in Duchenne (DMD) Research
Read More: dmdwarrior.com/zebrafish-mo...
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna #zebrafish #crispr #crisprcas9
dmdwarrior dmd warrior
WE WILL FIGHT DUCHENNE and WE WILL WIN 💪
👉 dmdwarrior.com
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna
No one cares about your child as much as you do. Together, we are stronger. 💪 dmd warrior
No one cares about your child as much as you do. Together, we are stronger. 💪
#dmd #duchenne #bmd #becker
#genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna
Muscular Dystrophy Association Announces Over $5 Million in Research Grants to Advance Neuromuscular Disease Research
Muscular Dystrophy Association Announces Over $5 Million in Research Grants to Advance Neuromuscular Disease Research
#dmd #duchenne #bmd #becker
#genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #musculardystrophy #gene #dna #mda
Novartis Buys Kate Therapeutics, Expanding in Neuro Gene Therapy
Novartis Buys Kate Therapeutics, Expanding in Neuro Gene Therapy
Novartis Buys Kate Therapeutics, Expanding in Neuro Gene Therapy
Novartis Buys Kate Therapeutics, Expanding in Neuro Gene Therapy
Source: www.novartis.com/stories/addr...
#dmd #bmd #genetherapy #dmdwarrior #dmdwarriors
#duchennemusculardystrophyawareness #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #novartis
Dr. Jason Pugh studies cognitive deficits in Duchenne muscular dystrophy
Dr. Jason Pugh studies cognitive deficits in Duchenne muscular dystrophy
Dr. Jason Pugh studies cognitive deficits in Duchenne muscular dystrophy
Dr. Jason Pugh studies cognitive deficits in Duchenne muscular dystrophy
Dr. Jason Pugh studies cognitive deficits in Duchenne muscular dystrophy 🧠 Research at UT Health San Antonio
Learn more: news.uthscsa.edu/trailblazing...
#dmd #genetherapy #dmdwarrior #dmdwarriors
#duchennewarriors #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #brain
Gower’s sign, or Gower’s movement, is a technique that makes getting up off the floor easier, and is most often indicative of proximal muscle weakness. It is one of a few common symptoms of Duchenne muscular dystrophy.
#duchennemusculardystrophy #duchenneawareness #79on79
