15th Annual Champions to CureDuchenne Returns to The University of Texas Golf Club on April 6 to Find a Cure for  Duchenne Muscular Dystrophy
CureDuchenne
by ashley
3d ago
 “Disco For Duchenne” Gala Features Dancing, Drinks, a Culinary Experience and More   to Help Advance Research for the Rare Disease  AUSTIN, Texas., February 28, 2024 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, and the Revell family of Austin will host the 15th annual fundraiser gala, Champions to CureDuchenne: Disco for Duchenne, on April 6, 2024. Austin’s own “Saturday Night Fever” will be filled with glittering lights and pulsating beats, bringing hundreds of philanthropists, businesspeople, and local commu ..read more
Visit website
CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients
CureDuchenne
by ashley
3d ago
Dyne Therapeutics will present initial clinical data, first released on January 3rd, 2024, from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Recorded Tuesday, February 13, 2024 Watch Recording The post CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients appeared first on CureDuchenne ..read more
Visit website
How to Identify and Manage Fat Embolism Syndrome (FES) in Fractures
CureDuchenne
by Andrea
2w ago
Watch the webinar HERE Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone fractures. It’s important to know the symptoms and what to do so that you can respond quickly. Check out CureDuchenne’s conversation with Dr. Brenda Wong, a neuromuscular physician from the Duchenne Clinic at the University of Massachusetts, and two parents with firsthand experience of FES to learn: The symptoms of FES Early intervention in FES How to communicate the risks with doctors who might be unfamiliar with FES Order a FREE CureDuchenne medical ID bracelet for y ..read more
Visit website
Finding the Right One: Advice from Experts on Dating with a Disability
CureDuchenne
by hawken@cureduchenne.org
3w ago
“So, are you dating anyone?” Could there possibly be five more scary words spoken to a single person? Finding love is hard for anyone and can be especially tricky if you have a disability, however, it is not impossible, says dating coach Erika Ettin. As Valentine’s Day approaches, we talked with Erika (@alittlenudge) and Ryan J Russell, PhD, a mindset coach and 41-year-old with Duchenne muscular dystrophy who was recently engaged, and developed five tips and tricks for finding success and experiencing worthwhile relationships for someone with a disability. 1. Be yourself!  Let’s be honest ..read more
Visit website
REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
CureDuchenne
by Andrea
3w ago
RegenXBio shared positive updates from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial for individuals with Duchenne aged 4-11 years old.  The 3 individuals in the lowest dose cohort all demonstrated reductions in CK levels, and expression of micro-dystrophin averaging 44% at 3 months.  Enrollment is also complete for a second cohort, which will receive a higher dose of the experimental therapy.  RegenXBio plans to share initial strength and functional data for both dose levels and initiate a pivotal trial in the second half of this year.  READ THE FULL PRE ..read more
Visit website
Keep Dreaming for the Future – Even with DMD
CureDuchenne
by hawken@cureduchenne.org
1M ago
By Jacob Gapko Jacob Gapko is 46 years old and has Duchenne muscular dystrophy. He uses a power wheelchair and non-invasive ventilation 24/7. He has a B.S. in physics and minors in mathematics and library science. Jacob currently serves as chief information officer for Family, Friends, and Duchenne. This blog post originally appeared on Family, Friends, and Duchenne. Where to begin—the easiest place to start is at the beginning. I was born November 15, 1977, in Eau Claire, WI, USA. I was diagnosed at age 3, and I have always known exactly what I have.  My uncle Howard also ..read more
Visit website
Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
CureDuchenne
by Andrea
1M ago
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and non-ambulatory individuals who received the high dose of 30 mg/kg once every 4 weeks had an average dystrophin expression of 5.17% at 28 weeks. This is significantly higher levels of dystrophin than seen with Sarepta’s FDA-approved agent, Exondys 51 (eteplirsen). Compared to Exondys 51, SRP-5051 contains a peptide designed to increase the ability of the exon-skipping agent to get ..read more
Visit website
CureDuchenne Welcomes Patricia Brown as Senior Director of Community Engagement 
CureDuchenne
by ashley
1M ago
Seasoned Healthcare Professional Brings Experience in Patient Advocacy and Community Education to Global Rare Disease Nonprofit  Newport Beach, Calif. – January 22, 2024 – CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, today announced an addition to its executive team with the hiring of Senior Director of Community Engagement, Patricia Brown, MBA, BSN, R.N. This position plays a crucial role in CureDuchenne’s mission of improving the lives of individuals and families affected by Duchenne muscular dystrophy, championing the needs o ..read more
Visit website
CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy  
CureDuchenne
by ashley
1M ago
Upcoming Events Across the Country Provide Latest Treatment Information and Resources for Managing Challenges of Living with Duchenne or Becker and Improving Quality of Life   NEWPORT BEACH, Calif., January 22, 2024 – CureDuchenne, a global leader in research, patient care and innovation for improving and extending the lives of those living with Duchenne muscular dystrophy, has announced its schedule of CureDuchenne Cares workshops and dinner sessions for the first half of the year, as well as its annual conference. CureDuchenne Cares provides interactive education and outreach programs t ..read more
Visit website
Protected: A Conversation With Justin Skeesuck From “I’ll Push You”
CureDuchenne
by hawken@cureduchenne.org
1M ago
This content is password protected. To view it please enter your password below: Password: The post Protected: A Conversation With Justin Skeesuck From “I’ll Push You” appeared first on CureDuchenne ..read more
Visit website

Follow CureDuchenne on FeedSpot

Continue with Google
Continue with Apple
OR