CureDuchenne

02/14/2026

Love takes many forms — and so does resilience. 💙

This Valentine’s Day, we’re celebrating couples living with Duchenne and the power of partnership, patience, and unwavering support.

Because Duchenne may shape the journey, but it will never define the love. ❤️

02/14/2026

Dating with a disability isn’t about limitations — it’s about communication, confidence, and finding someone who values you fully. 💙

As Valentine’s Day approaches, we thought it would be good to bring back the talk we had with Erika Ettin and Ryan J Russell, PhD, a mindset coach and 41-year-old with DMD. Together, they have developed five tips to help people with disabilities build meaningful relationships.

What tips would you add? Comment below!

Read the full blog using the link in the Linktree in our bio!

02/13/2026

Mark your calendars! 📅✨

Our 2026 CureDuchenne Workshops and Community Dinner Sessions dates are here. Join us for meaningful connections, expert insights, and time together as a community. We can’t wait to see you! 💙

Link to more information and registration: https://cureduchenne.org/events/category/community-events/

02/13/2026

💙💙💙

Ready to rock day two of Mate Show. Swing on by and pick up a consignment sheet for the sale Sept 17 at BLS.
CureDuchenne
Billings Livestock & Public Auction Yards
Cowboy Meat Company
KGHL
Western Ag Network
406 Precision
Western Ag Reporter
MH Equipment & Ag Resources

02/12/2026

We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the data are insufficient to meet the standard for approval. The company is expected to inform the community in the coming weeks of next steps for those currently receiving Ataluren therapy.

Our hearts are especially with the families who have participated in this clinical program for years—those who committed their time and endured countless visits in hopes of moving the science forward. We recognize how heavy this moment may feel.

We are deeply grateful to these families and to the team at PTC who dedicated years of work to advancing this research. Progress in Duchenne is only possible because of that commitment.

CureDuchenne stands with every family impacted, and we remain committed to accelerating promising research and expanding options so that no one with Duchenne is left behind.

Read the PTC press release and letter to the community.

For individuals with nonsense mutation Duchenne (about 10–15% of the community) this is incredibly difficult news. The need for effective, durable treatments remains urgent.

02/12/2026

Solid Biosciences shared that they have aligned with the US FDA on the overall design of a Phase 3 randomized, double-blind, placebo-controlled trial for SGT-003, a micro-dystrophin gene therapy for Duchenne. Solid also indicated that they will have additional meetings with the FDA regarding a potential accelerated approval pathway for SGT-003.

In the meantime, Solid’s Phase 1/2 trial is ongoing, with 36 participants dosed so far. The Company anticipates that Phase 3 will begin dosing before the end of Q1.

Read press release:

Solid Biosciences shared that they have aligned with the US FDA on the overall design of a Phase 3 randomized, double-blind, placebo-controlled trial for SGT-003, a micro-dystrophin gene therapy for Duchenne

02/12/2026

Satellos, a CureDuchenne funded company, just announced the first participant was dosed in their Phase 2 pediatric study of SAT-3247 for Duchenne muscular dystrophy.

Read the full news release:

First participant was dosed in their Phase 2 pediatric study of SAT-3247 for DMD.

02/11/2026

Today on International Day of Women and Girls in Science, we celebrate the remarkable women on our team who are leading innovation and turning scientific possibility into real progress for the Duchenne community.

Debra Miller, Founder and CEO, leads CureDuchenne by advancing high-impact research investments and advocating relentlessly to accelerate the development of transformative therapies for the Duchenne community.

Lianna Orlando, PhD, Vice President of Research, guides CureDuchenne’s scientific strategy, identifying high-potential research, advising strategic investments, and partnering with biotech leaders to accelerate therapies from discovery to clinical development.

Brenda Wong, MD, Chief Medical Advisor, shapes our clinical strategies by ensuring that every program and partnership reflects the highest standards of patient-centered care and therapeutic innovation.

Jennifer Wallace, PT, leads CureDuchenne’s Professional Education Program for Physical and Occupational Therapists, educating healthcare professionals around the world to elevate standards of care and guiding families on best-practice approaches.

Each of these leaders plays a distinct but powerful role in accelerating science and redefining care for the Duchenne community. Join us in celebrating these incredible women and all those focused on changing the future for those with Duchenne. share your appreciation and words of encouragement in the comments below!

02/11/2026

Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy. This clearance allows the company to move forward with a Phase 1/2 clinical study to evaluate safety and early signals of effectiveness.

The investigational therapy uses Precision’s ARCUS® gene editing platform and is designed to directly edit the dystrophin gene in muscle tissue. Notably, this program represents the first in vivo gene editing approach intended to potentially treat up to 60% of individuals living with Duchenne.

While this is an early step, FDA clearance to begin clinical testing marks important forward momentum for the Duchenne community.

Read the press release:

Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy.

02/11/2026

FUTURES 2026 here we come 💫 | May 21-24 | Orlando, Florida

In celebration of , Bionewsrare is awarding two $2,500 scholarships to
patients and caregivers to cover travel, lodging, and registration for the conference of their
choice. Which makes the perfect opportunity for our DMD families to have the chance to attend FUTURES in Orlando!

Apply by March 6 for the chance to be awarded the scholarship and join us for an unforgettable conference. Click the link below to read more and apply 💙

https://ow.ly/mlN050YcASf

02/10/2026

CureDuchenne is pleased to announce that it participated in the Satellos public offering, to support the company’s plans to advance the clinical development of SAT-3247. SAT-3247 is an orally administered experimental therapeutic aimed at addressing deficits in muscle repair and regeneration seen in Duchenne. If successful, this approach could be utilized by individuals with any type of Duchenne mutation.

"CureDuchenne continues to play an important role in advancing progress for the Duchenne community," said Frank Gleeson, Satellos co-founder and CEO. "Their support helps enable innovative approaches like ours as we work toward developing a new therapy for people living with Duchenne.”

Read our blog and the Satellos press release:

CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne Therapy

02/06/2026

FUTURES 2026, May 21-24 | Orlando, Florida

Cruising to Orlando for another great year together 💙

Register today at https://web.cvent.com/event/ee9e7402-2c59-420f-9cf9-5731cf20d4d9/summary