We are writing to provide some additional information to accompany our press release (attached) this week about the status of the rolling New Drug Application at the US Food and Drug Administration for vamolorone. As stated, Santhera met its target deadline of 6/28/2022, for submission of the vamorolone clinical module, and we want to acknowledge the efforts of our internal and partner teams for that great accomplishment.

However, as our press release on 6/29/22 indicated, the completion of the rolling NDA for vamorolone has been delayed due to the inspection readiness of a third-party contract manufacturing partner. At present, Santhera and the third-party contract manufacturing organization estimate that the delay will be 4-6 months. This new timeline pushes the NDA submission completion date for vamorolone to the fourth quarter of this year.

While we are disappointed by this delay, we want to take this opportunity to thank all of the patients and families who participated in the vamorolone studies, and to re-affirm our commitment to the vamorolone program and to the Duchenne patient community. We will continue to keep the community updated about the status of our NDA submission to the FDA.

The post Santhera Letter to the Community on NDA Filing for Vamorolone appeared first on CureDuchenne.

“With a commitment to supporting the Duchenne community with best practices in care until a
cure is found, we are thrilled to offer the online DME Guide to families,” said Debra Miller,
founder and CEO, CureDuchenne. “While parents and caregivers are determined to find the
equipment that will help keep their loved ones active in any way possible, we are determined
to help them make the best decisions by providing them with all the resources they need in one
place.”

As the progression of Duchenne begins to rob individuals of muscle strength and ambulation,
mobility equipment can offer realistic means for those with Duchenne to maintain participation
in daily living activities and preserve as much independence as possible. CureDuchenne’s online DME Guide will be updated regularly to provide current information on equipment utilized by individuals living with Duchenne. The site includes information on mobility devices, orthoses, home equipment and respiratory devices. Additionally, the site features helpful links and downloadable information about equipment and proper use, and educational videos about equipment use produced by CureDuchenne.

About CureDuchenne
CureDuchenne is the leading nonprofit dedicated to extending and improving the lives of
300,000 boys worldwide who have Duchenne muscular dystrophy, a degenerative disease with
no known cure. CureDuchenne uses an entrepreneurial venture philanthropy model to fund
impactful research and accelerate human clinical trials. Through its CureDuchenne Cares program, CureDuchenne provides confidence to families seeking valuable resources, trains
physical therapists and other health professionals on new standards of care and provides hope,
support and empowerment to the Duchenne community. For more information, please visit
www.cureduchenne.org.

The post CureDuchenne Helps Duchenne Families Make Informed Decisions About Durable Medical Equipment with New Interactive Website appeared first on CureDuchenne.

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The post Erin Frey to Lead CureDuchenne’s DMD Advocacy Efforts appeared first on CureDuchenne.

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Lucas was 5 before his parents, Bill and Marci Barton of Grand Haven, Mich., finally got an explanation for his difficulties for standing up or climbing stairs. The diagnosis: muscular dystrophy.

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The post For Many Boys With Duchenne Muscular Dystrophy, Bright Hope Lies Just Beyond Reach appeared first on CureDuchenne.

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“Out motto is ‘leave no boy behind,’ so we want to make sure we have a format that works in smaller, less populated markets,” Debra Miller, CureDuchenne’s co-founder and CEO, told Muscular Dystrophy News in a phone interview.

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The post CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference appeared first on CureDuchenne.

If you missed the movie, you may have caught wind of He Jiankui. Near the end of 2018, the Chinese scientist reported having used CRISPR to produce the first-ever gene-edited babies. (He currently faces criminal charges in his home country for violating national guidelines, not to mention global scientific ethical standards.)

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The post Why My Patient Advocacy Organization Is Investing In CRISPR appeared first on CureDuchenne.

A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at age 6 and immediately began taking corticosteroids as well as heart medicine and supplements.

“I’ve been well-medicated all my life, so I’m fine,” the 33-year-old said in a phone interview. “I’m in good health.”

It also helps that both his parents, David Feder and Ana Lucia Langer, are doctors.

“When I was diagnosed with Duchenne, there wasn’t much information about the disease, so they decided to research it by themselves,” he said. “My father is a pulmonologist and general clinician. And my mother, originally a pediatrician, now only treats neuromuscular diseases.”

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The post Defying the Odds of Living with Duchenne, Decade After Decade appeared first on CureDuchenne.

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