Read Avidity’s Community Letter:

The post Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027 appeared first on CureDuchenne.

As an early funder of Entrada Therapeutics, we are pleased to share their latest newsletter to the Duchenne community.  This update includes news that after review of all the data in the first dose (5 mg/kg) cohort of the ELEVATE-45-201 study for individuals amenable to skipping exon 45, an independent data monitoring committee has recommended dose escalation up to 10mg/kg for a second cohort.   The company is on track to report data from Cohort 1 in mid-2026.

Link to community newsletter: https://us8.campaign-archive.com/?u=17d71fdc6c0bc448a2c3a260c&id=87546b3f50

Link to press release: https://ir.entradatx.com/news-releases/news-release-details/independent-safety-data-monitoring-committee-recommends

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CureDuchenne invested in Edgewise in 2019, recognizing the potential of their approach. This acquisition is a powerful validation that CureDuchenne’s venture philanthropy approach works, helping accelerate promising science, attract investment, and advance potential therapies toward patients. 

A Significant Step Forward for Becker and Duchenne Muscular Dystrophy 

Sevasemten, which aims to limit the muscle damage caused by the absence or loss of functional dystrophin, is in late-stage clinical trials in both Becker and Duchenne muscular dystrophy.   

In Becker muscular dystrophy (BMD), topline data from the pivotal cohort of the GRAND CANYON study is expected in the fourth quarter of 2026.  Participants in the Phase 2 open label extension studies have seen stabilization of functional measurements, in marked contrast to the decline reported in BMD natural history studies.   If successful, sevasemten will become the first and only approved therapy specifically for individuals living with BMD. 

Edgewise has also reported positive Phase 2 studies with sevasemten in patients with Duchenne.  The progress of both of these programs represents years of scientific innovation and collaboration aimed at addressing significant unmet medical needs. 

For families affected by Becker and Duchenne muscular dystrophy, today’s announcement reinforces confidence that the programs will continue advancing toward potential regulatory approval and commercialization. 

Validation That CureDuchenne’s Model Works 

At CureDuchenne, we have long championed a venture philanthropy approach by investing in promising science, supporting innovative biotechnology companies, and helping de-risk programs that have the potential to transform the lives of individuals living with Duchenne and Becker muscular dystrophy. 

The acquisition of the Edgewise neuromuscular portfolio by a major global pharmaceutical company demonstrates that this model is working. This is exactly the type of progress our community has worked toward for decades. 

Congratulations to the Edgewise Team 

We extend our sincere congratulations to the entire Edgewise Therapeutics team for reaching this important milestone. Their commitment to developing therapies for Becker and Duchenne muscular dystrophy has helped advance the field and bring new hope to families worldwide. 

We also welcome Servier to the Becker and Duchenne communities and look forward to working alongside their team as these programs continue to advance. 

Looking Ahead 

While important milestones remain ahead, today’s announcement reflects growing momentum across the neuromuscular disease landscape. CureDuchenne remains committed to accelerating therapies, improving care, and ensuring that every promising opportunity reaches its full potential for the benefit of patients and families around the world. 

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In parallel, Dyne has launched a Phase 3 confirmatory trial of z-rostudirsen, and is advancing 4 additional exon-skipping programs for exons 53, 45, 44 and 55.     

Link to press release:  https://www.globenewswire.com/news-release/2026/05/26/3300969/0/en/dyne-therapeutics-announces-submission-of-biologics-license-application-bla-to-u-s-fda-for-z-rostudirsen-in-exon-51-duchenne-muscular-dystrophy-dmd.html

Community letter:  https://www.dyne-tx.com/forzetto-trial-initiated/

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Dr. Gardner most recently served as Senior Medical Director, Global Medical Affairs – Neurology & Immunology at EMD Serono, a business of Merck KGaA, Darmstadt, Germany. In that role, she led global medical strategy for neuroimmunology programs and collaborated across clinical development, regulatory, real-world evidence, and commercial teams. Prior to joining EMD Serono, she worked in Medical Affairs and clinical development at Novartis, overseeing spinal muscular atrophy and other clinical trials. Earlier in her career, Dr. Gardner served as an Associate Professor of Neurology at the University of Tennessee Health Science Center, where she built and led a translational research program focused on neuroinflammatory and neuromuscular diseases.

As Vice President of Clinical and Medical Affairs, Dr. Gardner will provide strategic medical leadership across CureDuchenne’s research, education, and patient care initiatives. She will work closely alongside CureDuchenne’s Chief Medical Advisor, Brenda Wong, MD, an internationally recognized Duchenne expert, to help advance standards of care, strengthen educational resources, and accelerate therapeutic development for individuals and families affected by Duchenne and Becker muscular dystrophy. Dr. Gardner will also serve as a key spokesperson for CureDuchenne within the medical and scientific communities and help drive strategic partnerships that support the organization’s mission and long-term impact.

“Lidia brings an exceptional combination of scientific expertise and deep commitment to patient-centered care,” said Debra Miller, founder and CEO of CureDuchenne. “Her experience building strong relationships across industry, advocacy, and clinical communities will be instrumental as we continue accelerating research, advancing standards of care, and ensuring families have access to trusted education and support. Lidia understands the urgency facing our community and shares our commitment to driving meaningful progress for everyone affected by Duchenne.”

“I am honored to join CureDuchenne and contribute to an organization that has played such a transformative role in advancing research and care for the Duchenne community,” said Dr. Gardner. “I look forward to partnering with families, clinicians, researchers, and industry leaders to help accelerate therapies, strengthen education and support programs, and continue building momentum toward a future where every individual with Duchenne has access to better care and more treatment options.”

About CureDuchenne
Over twenty years ago, CureDuchenne was created with one goal: to find and fund a cure for Duchenne muscular dystrophy, one of the most common and severe forms of muscular dystrophy. Today, CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne. CureDuchenne’s innovative venture philanthropy model has advanced transformative treatments for Duchenne muscular dystrophy, investing more than $27 million in early-stage research that helped move 19 programs into human clinical trials. For more information on how to help raise awareness and funds needed for research, please visit cureduchenne.org or follow us on facebook, Instagram, LinkedIn, and X.

The post CureDuchenne Appoints Lidia Gardner, PhD, EMBA, as Vice President of Clinical and Medical Affairs to Advance Patient-Centered Care, Research, and Strategic Partnerships appeared first on CureDuchenne.

Newport Beach, Calif., May 20, 2026 —CureDuchenne, a global leader in funding and advancing research for Duchenne muscular dystrophy, today announced a second investment into Tevard Biosciences to support the advancement of the company’s suppressor tRNA (suptRNA) therapy platform for Duchenne muscular dystrophy caused by nonsense mutations. Nonsense mutations encode a premature stop codon that halts translation and prevents formation of a functional full-length protein.

CureDuchenne first invested in Tevard Biosciences in 2023 after recognizing the company’s mutation-specific approach to addressing a significant unmet need in the Duchenne community. Since then, CureDuchenne has continued to support Tevard’s work as the company has advanced its platform and generated encouraging preclinical data demonstrating restoration of full-length dystrophin protein in Duchenne disease models.

For the first time, Tevard Biosciences will present its most recent data directly to the Duchenne community at the CureDuchenne FUTURES National Conference on May 22, 2026. The presentation will provide families, clinicians, researchers, and advocates an opportunity to learn more about the company’s progress and suppressor tRNA approach for nonsense mutation Duchenne. CureDuchenne will also host a webinar with Tevard Biosciences on Thursday, May 28, 2026 at 12 p.m. PT. Webinar registration is now open.

Tevard’s suppressor tRNA technology is designed to address nonsense mutations, which affect approximately 12% of individuals living with Duchenne. Tevard’s approach is mutation-specific and aims to restore production of full-length, natural dystrophin protein by enabling the cell’s normal machinery to read through premature stop codons. Currently, there are no approved therapies specifically designed to treat this class of Duchenne mutations, leaving many families with limited treatment options and highlighting the urgent need for continued innovation in this area.

Recently released preclinical data from Tevard demonstrated restoration of an average of 70% of wild-type dystrophin protein levels in Duchenne preclinical models using the company’s latest-generation suppressor tRNA candidates, along with functional improvements and durable protein expression following a single intravenous dose.

“We are deeply grateful for CureDuchenne’s early belief in our platform and their continued partnership as we advance this important work,” said Daniel Fischer, Co-Founder, President and CEO of Tevard Biosciences. “CureDuchenne recognized the urgent unmet need facing individuals with nonsense mutations and understood the potential of suppressor tRNA technology to restore full-length, natural dystrophin protein. Their funding, scientific guidance, and commitment to the Duchenne community have helped accelerate our progress as we work to bring forward a potential new treatment option for families who urgently need one.”

“For families affected by nonsense mutation Duchenne, there remains a critical unmet need for therapies designed specifically for this mutation class,” said Debra Miller, founder and CEO of CureDuchenne. “We saw the potential in Tevard’s technology early and continue to be encouraged by the potential of Tevard’s approach to restore full-length dystrophin protein and expand treatment possibilities for individuals who currently have very limited options. CureDuchenne is proud to support innovative science like this, and we welcome other organizations and investors to join us and help advance promising approaches that could bring more treatments to more people faster.”

About CureDuchenne
Over twenty years ago, CureDuchenne was created with one goal: to find and fund a cure for Duchenne muscular dystrophy, one of the most common and severe forms of muscular dystrophy. Today, CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne. CureDuchenne’s innovative venture philanthropy model has advanced transformative treatments for Duchenne muscular dystrophy, investing more than $27 million in early-stage research that helped move 19 programs into human clinical trials. For more information on how to help raise awareness and funds needed for research, please visit cureduchenne.org or follow us on facebook, Instagram, LinkedIn, and X.

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Link to the press release:   here

The post Dyne initiates global Phase 3 trial in Duchenne amenable to skipping exon 51, ahead of planned FDA submission for accelerated approval in the US appeared first on CureDuchenne.