Uncategorized

CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a black box warning in the ELEVIDYS label, resolving any material issues with the ambulant population indication. Sarepta has completed an Expert Committee on a protocol for the…

Know More

Mike Huddleston is 62 years old. He is originally from California and now lives in Maryland.  Mike was diagnosed with spinal muscular atrophy (SMA) Type 3 at the age of 16. He started using a power wheelchair in 2015.  He and his wife of over 40 years, Debbie, are both retired. Mike volunteers with several organizations…

Know More

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease, following the agency’s decision to not accept the original filing. According to a company press release, the FDA issued a complete response letter (CRL) requesting additional clinical data, stating…

Know More

July is my favorite month. Many people who know me might be surprised by that, as they’re aware that I’m a Christmas enthusiast who loves to decorate, making my house look like the North Pole for nearly eight weeks every winter. I spend the other 10 months of the year thrifting for the perfect Christmas…

Know More

Capricor Therapeutics announced that it has received a Complete Response Letter indicating that the FDA has completed the review of Deramiocel for Duchenne cardiomyopathy, but would be unable to approve the application in its current form.  Capricor shared that the letter cites that their application does not meet the statutory requirement for substantial evidence of…

Know More

Lorraine Woodward, founder and CEO of Becoming rentABLE. Expedia Group is not only opening the door, but widening the doorway, when it comes to accessible short-term rentals (STR) and vacation homes. As one of the largest travel companies in the world, hosting travel metasearch engines like Vrbo, Tavelocity, and more, the initiative to increase accessible…

Know More

On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means that the current application will not be approved in its present form, but it also presents a path forward by…

Know More

As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials.  With all therapies, there are associated side effects and it’s critical that families and individuals who receive gene therapy (or any therapies) understand…

Know More

Nearly one-third of people with muscular dystrophy (MD) experience moderate pain, which varies by the type of their MD, a study with more than 1,200 participants suggests. To manage their pain, about a fifth of patients used prescribed pain medications, mostly non-opioid therapies at first, then combined with opioids over time. Impaired mobility was the…

Know More

Need direct support for yourself or your child living with dystrophinopathy? PPMD’s PPMD For You program is here to help! This personalized program lets you schedule a one-on-one meeting with a knowledgeable PPMD team member to access individualized resources in the areas of care, research, and daily life. Whether you’re looking for guidance, answers, or just…

Know More