Center for Duchenne Muscular Dystrophy at UCLA

02/17/2026

We are sorry to share this news and send our sincerest gratitude to all the patients and families who participated in the research and clinical trials. Your time and sacrifice mattered.
Feb. 12, 2026
Update to the U.S. Duchenne Community on the Ataluren (Translarna™) NDA Review:
Recent discussions with the U.S. Food and Drug Administration (FDA) have made clear that
there are differences in data interpretation that cannot be successfully resolved to enable
approval of ataluren (Translarna™) for the treatment of nonsense mutation Duchenne muscular
dystrophy in the U.S. Despite the evidence of safety and effectiveness demonstrated across
several clinical studies, FDA has shared that they view the data as insufficient to meet their
threshold for approval. Accordingly, PTC has made the difficult decision to withdraw the
resubmission of the New Drug Application for ataluren.
We understand that this outcome is devastating for the Duchenne muscular dystrophy
community, the families who have participated in clinical trials for almost 20 years, and the
families who continue to wait for a treatment option that addresses the underlying cause of
nonsense mutation Duchenne muscular dystrophy.
Over the coming weeks, we will be determining next steps regarding supply of ataluren for those
currently receiving therapy. We will provide more information once we complete our
assessment. If you have any questions, please feel free to reach out to our patient engagement
team at patientengagement@ptcbio.com.
We are humbled by the community's commitment and courage and deeply appreciate the many
years of steadfast support in our shared mission. We are disappointed that, despite our best
efforts, we have not been able to achieve FDA approval for ataluren.
https://www.ptcbio.com/
PDF of community Letter here:
https://www.ptcbio.com/wp-content/uploads/sites/2/2026/02/PTC-Update-to-the-Duchenne-community-Feb-2026.pdf

Translating Science. Transforming Lives.

02/12/2026

February 12, 2026
Satellos Announces First Participant Dosed in Phase 2 Pediatric Study of SAT-3247 for Duchenne Muscular Dystrophy

BASECAMP, a three-month, randomized, double-blind, placebo-controlled study, will evaluate SAT-3247’s safety and tolerability, and effect on muscle force, muscle quality and muscle regeneration.
Full PR here:

BASECAMP, a three-month, randomized, double-blind, placebo-controlled study, will evaluate SAT-3247’s safety and tolerability, and effect on muscle force, muscle quality and muscle regeneration Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a clinical-sta...

02/12/2026

February 11, 2026
Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dystrophy

– U.S. FDA Study May Proceed notification enables initiation of clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical study in patients with Duchenne muscular dystrophy (DMD) –

– Institutional Review Board (IRB) process is underway at multiple world-class DMD clinical trial sites –

– PBGENE-DMD is an in vivo gene editing investigational product designed to correct the underlying genetic cause of DMD through a novel one-time gene editing therapy –

– Company plans to host a virtual event in March after the Muscular Dystrophy Association conference to discuss PBGENE-DMD and the FUNCTION-DMD clinical study with participation from key opinion leaders including patient advocacy groups.
Full PR here:
https://investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-receives-us-fda-clearance-investigational

02/09/2026

February 9, 2026 Solid Biosciences Announces Positive Feedback from Type C Meeting with FDA for SGT-003 Gene Therapy for Duchenne Muscular Dystrophy.

- IMPACT DUCHENNE: Company aligned with FDA on Phase 3 randomized, double-blind, placebo-controlled trial design -

- IMPACT DUCHENNE: Company anticipates first participant dosing in Q1 2026 -

- Company plans for additional meetings with the FDA in 1H 2026 to align on a potential accelerated approval pathway for SGT-003

- INSPIRE DUCHENNE: SGT-003 continues to be generally well tolerated with 36 participants dosed as of February 9, 2026, in Phase 1/2 trial -

full PR here:
https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-positive-feedback-type-c-meeting-fda

02/05/2026

https://www.prnewswire.com/news-releases/cumberland-pharmaceuticals-receives-fda-fast-track-designation-for-its-ifetroban-duchenne-muscular-dystrophy-program-302678893.html

/PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases,...

02/03/2026

Breaking news from EveryLife Foundation!
February 3, 2026
Congress Passes Five-Year Reauthorization of Rare Pediatric Disease PRV Program!

Thank you to all the organizations and Rare Disease advocates who helped make this happen!

"Today, our rare disease community has a reason to celebrate, as Congress passed legislation that includes a five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, increased funding for rare disease research, and other healthcare priorities that matter deeply to patients and families. "

01/29/2026

Rare Disease Week on Capitol Hill. Save the Date: February 24- 26, 2026
This free multi-day event, hosted by the Rare Disease Legislative Advocates, a program of the EveryLife Foundation for Rare Diseases, brings together rare disease advocates from across the country to make their voices heard with their Members of Congress. Participants are educated on policy proposals impacting the rare disease community and provided opportunities to advocate directly for policy change with their Members of Congress.

Rare Disease Week - EveryLife Foundation for Rare Diseases

01/26/2026

PPMD will host a webinar today with Sarepta Therapeutics to discuss the newly released 3 year top line data for Elevidys. Register here for the webinar TODAY (January 26, 2026) at 1pm PT.

Join PPMD and Sarepta Therapeutics for a breaking news community webinar on Monday, January 26, 2026, at 4:00 PM ET to discuss the recently released three-year top line data for ELEVIDYS, Sarepta’s gene therapy for the treatment of Duchenne. The presentation will include a review of the data, a ti...

01/23/2026

Please join PPMD for a community webinar with Dyne Therapeutics on Wednesday, January 28th at 1:00 PM ET. The webinar will feature a presentation with Dr. Doug Kerr, Dyne’s Chief Medical Officer, who will share topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits out to 24 months.
Register here:
https://www.parentprojectmd.org/events/webinar-dyne-therapeutics-topline-results-from-phase-1-2-deliver-trial/?fbclid=IwY2xjawPg2uZleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEe_hTphWSuVSmb2W7fevlzjnW_pdNzhfGFPkbEei5dkdZ936-Y-Fgd2668ORo_aem_AOWQutdEUJOkIv295I-DTw

Please join us for a community webinar with Dyne Therapeutics on Wednesday, January 28th at 1:00 PM ET. The webinar will feature a presentation with Dr. Doug Kerr, Dyne’s Chief Medical Officer, who will share...

01/23/2026

On Monday Jan. 26 at 8:30am Eastern Time, Sarepta will share 3 year topline results from the Embark Phase 3 Study of Elevidys.

"Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Jan. 26, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present 3-year topline functional results from patients treated in Part 1 of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment."
Register Full PR here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-report-3-year-topline-data-embark-study

01/21/2026

Action Alert from PPMD! Urge Congress to support FY 2026 Duchenne funding. Click link and send emails to your representatives:
https://engage.parentprojectmd.org/component/sbxactions/36?fbclid=IwY2xjawPeNLBleHRuA2FlbQIxMABicmlkETF2ZTdraW9xRzBudXMzaEtkc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHsGOjZcINI3JmX9G85lQVH7vWYtqHl5MNmLuWeiZspF4uZ5Dh1uRJ43YoLRg_aem_DT61k9xlaEh4GFzNC0GQbg

Send a message to your Members of Congress urging them to support our priorities