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
CureDuchenne
Non-profit Organizations
Newport Beach, CA 5,059 followers
Together, we WILL cure Duchenne!
About us
CureDuchenne is the global leader in Duchenne research, patient care and innovation. We are committed to improving the lives of those affected by Duchenne through accelerating research, improving care and empowering the community. We won't stop until everyone has a cure. cureduchenne.org Founded by Paul and Debra Miller in 2003, after their son was diagnosed with the disease, CureDuchenne combines fundraising and venture philanthropy leveraging donor dollars to maximize support for promising research into effective treatments for those suffering from Duchenne. With transparency as a core value, investment proceeds are redeployed to support research and other mission critical programs to find a cure.
- Website
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http://www.cureduchenne.org
External link for CureDuchenne
- Industry
- Non-profit Organizations
- Company size
- 11-50 employees
- Headquarters
- Newport Beach, CA
- Type
- Nonprofit
- Founded
- 2003
- Specialties
- Research, Duchenne, DMD, Duchenne muscular dystrophy, Venture philanthropy, Venture Capital , Philanthropy, fundraiser, Community, Clinics, Support, and clinical trials
Locations
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Primary
100 Bayview Circle
Suite 5600
Newport Beach, CA 92660, US
Employees at CureDuchenne
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Vinh Ha
Director of Web Services | LAMP Developer (Linux, Apache, MySQL, PHP) | WordPress + WooCommerce Developer | UI/UX (Frontend) Developer/Designer |…
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Paul Miller
President and Chief Operating Officer at PMG
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Debra Miller
Chief Executive Officer/Founder at CureDuchenne
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Laura Hameed
Possibilitarian. Nonprofit Executive, U of MN Regent Emeritus, Board Director, Aspen Institute Rodel Fellow. Accelerating equitable access to…
Updates
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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
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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
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CureDuchenne reposted this
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
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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
Duchenne muscular dystrophy: Exercise and physical therapy –...
https://musculardystrophynews.com
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WEBINAR:: The FDA Approval of Sarepta Therapeutics's ELEVIDYS to Duchenne Patients Ages 4 and up. https://lnkd.in/g773j3kG #DMD #FDA #CureDuchenne #Duchenne
FDA Decision on Sarepta's ELEVIDYS Webinar
https://www.youtube.com/
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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
Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial
https://cureduchenne.org
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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
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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
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US FDA approves expanded use of Sarepta's Duchenne gene therapy; shares jump #stockmarket #FDA #DMD #Duchenne
US FDA approves expanded use of Sarepta's Duchenne gene therapy; shares jump
reuters.com