December 13, 2019

FDA Grants Accelerated Approval to Vyondys 53

PPMD is excited to learn that the FDA has granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne patients who have a confirmed mutation of the dystrophin gene that is amenable to exon…

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December 12, 2019

FDA Authorizes First Test to Aid in Newborn Screening for Duchenne

Today, the FDA authorized the marketing of the first test to aid in newborn screening for Duchenne. As you know, PPMD has been leading the effort for newborn screening in Duchenne. In October, the first…

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December 12, 2019

“PPMD makes my son’s life better.”

When our son Seth was diagnosed, my husband Chris and I took very different approaches. He wanted to find out everything he could about Duchenne whereas I didn’t think I could handle any more information….

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December 6, 2019

Donate to PPMD today, make tomorrow stronger

Thank you to everyone who donated to PPMD on #GivingTuesday! Your generosity and a matching gift from my dear friends, Mary-Lou & Larry Weisman, enabled PPMD to begin the holiday season strong. We hope you…

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December 4, 2019

Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics

Astellas Pharma and Audentes Therapeutics announced that they have entered into a definitive agreement for Astellas to acquire Audentes. We look forward to learning more as this partnership moves forward. Read the Announcement from Audentes…

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November 27, 2019

Thankful that you believe in PPMD

Today is one of my favorite days of the year. Thanksgiving is a holiday that revolves solely around friends and family coming together to just eat, maybe have a little wine, and then eat some…

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November 26, 2019

WATCH: NS Pharma Provides Viltolarsen Update – Webinar Recording

On November 20, 2019, NS Pharma joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of its investigational drug, viltolarsen (NS-065/NCNP-01), including its mechanism of action and regulatory status….

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November 19, 2019

Santhera shares SIDEROS clinical trial and BreatheDMD program update

Santhera Therapeutics shared a community letter today announcing that the company has decided to suspend enrolling new participants in the BreatheDMD expanded access program (EAP) until their SIDEROS clinical trial is fully enrolled. The SIDEROS…

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November 18, 2019

Finding Hope, Seeing Progress

Clinical trials are HARD. How many times have you heard that? But it’s true. Expectations, anticipation, screening, burden of participation with frequent clinic visits, blood draws, outcome measures and waiting for news. This year has…

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November 13, 2019

Catabasis Shares November 2019 Newsletter

Catabasis shares the latest edition of their community newsletter, including information about the role of NF-kB in muscle and an update about edasalonexent in development for the treatment of Duchenne. Click here to download (PDF).

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