February 20, 2019

PPMD Contributes to Clinical Practice Recommendations for Managing Adrenal Insufficiency Risk in Duchenne

Long term glucocorticoid (steroid) therapy has many clear benefits such as improving muscle strength and maintaining cardiac and pulmonary function. However, prescribed steroid (prednisone, Emflaza, or deflazacort) regimens need to be followed closely to avoid…

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February 19, 2019

End Duchenne Tour Recap: Las Vegas, NV

PPMD was thrilled to head to Las Vegas, NV for our first End Duchenne Tour stop of 2019 on February 2. More than 45 people came together to learn about the latest information and resources for Duchenne…

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February 19, 2019

Catabasis Presents Data Supporting Edasalonexent as a Potential Foundational Treatment for Duchenne

Catabasis Pharmaceuticals, Inc. shared additional clinical results from the MoveDMD trial of edasalonexent at the XVII International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy this weekend. We are pleased to learn that in…

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February 15, 2019

WATCH: Caring for the Caregiver – Webinar Recording

Caring for a child with a complex condition such as Duchenne or Becker muscular dystrophy can be a tremendous burden on caregivers, especially if those caregivers are family members. Not only is there an emotional…

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February 14, 2019

Sarepta Announces FDA Acceptance of Golodirsen NDA for People with Duchenne Amenable to Exon 53 Skipping

Sarepta Therapeutics, Inc. announced the FDA has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019. Golodirsen is a potential exon skipping…

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February 12, 2019

WATCH: Vamorolone Clinical Trials in Duchenne – Webinar Recording

On January 30, 2019, ReveraGen joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of the vamorolone drug development program for Duchenne, including results showing improved muscle function in…

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February 11, 2019

PPMD Designates University of Rochester Medical Center as a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named the University of Rochester Medical Center (URMC) the 24th Certified Duchenne Care Center. This expanding program of elite centers, created in 2014 as part of PPMD’s Certified Duchenne Care Center Program, reviews and…

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February 8, 2019

How Do I Find a Clinical Trial That’s Right for Me?

Are you getting confused by all the news and social media posts about clinical trials? Not sure which trials or studies may be appropriate for you or your child? You are not alone! Even healthcare…

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February 7, 2019

Solid Biosciences Announces Intention to Dose Escalate in IGNITE DMD Clinical Trial

Solid Biosciences announced today plans to move to a higher dose of SGT-001 in IGNITE DMD, their Phase 1/2 clinical trial evaluating SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. This decision is…

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February 6, 2019

NS Pharma Begins Rolling NDA Submission to FDA for Viltolarsen (NS-065/NCNP-01)

PPMD is pleased to learn that NS Pharma has initiated submission of a rolling New Drug Application (NDA) seeking accelerated approval for viltolarsen (NS-065/NCNP-01), an investigational drug for the treatment of Duchenne in patients amenable…

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