CureDuchenne

Join us for a full-day event focused on improving the quality of life for individuals living with Duchenne. PRESENTATIONS INCLUDE: Standards of Care | Family Support | Physical Therapy & Medical Equipment | Research & Clinical Trials | Open House. Venue: The Westin Minneapolis Address: 88 South 6th Street, Minneapolis, Minnesota 55402

Nathan, a talented student from The University of Texas at Austin, has been accepted into the Austrian American Mozart Academy in Salzburg! Despite the challenges of living with Duchenne muscular dystrophy, Nathan has secured a leading role in an opera during the academy. This opportunity is a dream come true! However, his travel and accommodations are costly due to his care needs. He's raising $15,000 to help make this journey. Every bit helps! Let’s support Nathan in achieving his dreams. Please help Nathan: https://lnkd.in/grrNW3b6 #DMD #CureDuchenne #MozartAcademy #DreamBig #Fundraiser

Join Us for FUTURES National Family Conference 2025 in San Antonio, Texas – A Transformative Event for the Duchenne Community! Registration is now open: https://lnkd.in/g7CSkdU2 #CureDuchenne #duchenne #DMD #sanantonio #duchenneCommunity. #FuturesForAll

Wow. What a week our community had last week. By now, you’ve probably heard the momentous news that the FDA broadened the label for ELEVIDYS gene therapy to include older boys with Duchenne, ages 4 and up. As both the mom of an incredible man with Duchenne and an advocate for all boys and men with this disease that takes abilities away from our loved ones every day, this milestone resonates deeply with me. For families like ours, who have faced the relentless challenges of Duchenne, this moment is a testament to the progress we are making. All boys and young men, like my son Hawken, urgently need treatments to slow the progression of this devastating disease. We do not have the luxury of time to wait for a perfect solution. With limited or no options available for so many, any treatment that can help delay the disease's progression is a huge step forward. The ability to choose gene therapy provides a critical option for many families and their physicians to consider, offering a glimmer of hope where there was little before. I’m thankful to the companies, like Sarepta Therapeutics, that are driving treatments forward, and to the FDA for advancing these innovative treatments. And our community stands in eternal gratitude to the families who have bravely participated in clinical trials, knowing the risks and taking them on anyways on behalf of their child and thousands of others who stand to benefit. But we’re not done yet. There are limitations to this treatment that we must address, and individuals who will still be excluded from treatment. We are doubling down on our efforts with renewed confidence and determination. We have come so far, and with the wind at our backs, we know we will cure this disease. CureDuchenne

Thank you, Muscular Dystrophy News, for this valuable resource on physical therapy in Duchenne muscular dystrophy featuring insights from our very own Physical Therapist, Jennifer Wallace. Read more here: https://lnkd.in/ge9EQHzD

WEBINAR:: The FDA Approval of Sarepta Therapeutics's ELEVIDYS to Duchenne Patients Ages 4 and up. https://lnkd.in/g773j3kG #DMD #FDA #CureDuchenne #Duchenne

Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe. The three participants receiving the study’s higher dose (3x1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage. Genethon said it is planning a pivotal trial in Europe. #CureDuchenne #duchenne #DMD #wewillcureduchenne

REGENXBIO Inc. announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory boys with Duchenne aged 4 to 11, is now expected to enroll up to five individuals aged 1-3 as well. These younger participants will receive RGX-202 at the pivotal dose level (dose level 2, at 2x1014 genome copies/kg body weight). REGENXBIO expects to share initial strength and functional assessment data for individuals already treated (at both dose levels) later this year. Press Release here: https://lnkd.in/grTuFBRi

Entrada Therapeutics, which received early funding from CureDuchenne, reported positive data from a Phase 1 ENTR-601-44-101 trial in healthy volunteers. ENTR-601-44, which is designed to skip exon 44, was well-tolerated, and the six individuals in the highest dose cohort (6 mg/kg) demonstrated an average of 0.44% exon skipping after a single dose. This was statistically significant compared to placebo control. Entrada plans to submit regulatory applications in Q4 2024 to initiate separate global Phase 2 clinical trials of ENTR-601-44 for Duchenne amenable to skipping exon 44, and for ENTR-601-45 for Duchenne amenable to skipping exon 45. They also plan to submit regulatory applications in 2025 for a global Phase 2 for Duchenne amenable to skipping exon 50. Link to press release: https://lnkd.in/gnd7kvUN

US FDA approves expanded use of Sarepta's Duchenne gene therapy; shares jump #stockmarket #FDA #DMD #Duchenne