August 7, 2019

Audentes Therapeutics Announces Plans to Commence Patient Dosing with AT702 in Q4 2019

Audentes Therapeutics has announced plans to commence patient dosing in the fourth quarter of 2019 with AT702 produced by Nationwide Children’s Hospital. AT702 is designed to induce exon 2 skipping to treat Duchenne caused by…

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August 1, 2019

Race Athlete Spotlight: Tara Reynolds

Tara Reynolds has run the Aramco Houston Half Marathon with the Race to End Duchenne team for three years in a row, raising thousands of dollars. She does it all for her friend and inspiration,…

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July 31, 2019

PTC Therapeutics Update on Specialty Pharmacy Transition

PTC Therapeutics has shared an update regarding the transitioning of patients that are taking EMFLAZA® (deflazacort) to a new specialty pharmacy, Accredo. Read the Update from PTC: PTC Therapeutics Update on Specialty Pharmacy Transition In…

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July 25, 2019

Newborn Screening Saves Lives Reauthorization Act Passes in House of Representatives

We are thrilled to share that last night the House of Representatives passed HR 2507 – the Newborn Screening Saves Lives Reauthorization Act by voice vote! PPMD has been proud to be a part of…

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July 24, 2019

ICER Roundtable – What Does It Mean?

In December 2018, the Institute for Clinical and Economic Review (ICER) announced that it would be conducting an assessment of three Duchenne products over the next seven months. The ICER assessment is culminating in a…

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July 19, 2019

WATCH: Capricor’s HOPE-2 Interim Analysis – Webinar Recording

On Wednesday, July 17, 2019, Capricor joined Parent Project Muscular Dystrophy for a webinar to update the community on Capricor’s HOPE-2 clinical program, following the company’s announcement of positive results from their interim analysis in…

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July 18, 2019

Roche/Genentech Announces Completed Recruitment for SPITFIRE Study

Roche/Genentech has provided an update to the community announcing that enrollment in the SPITFIRE pivotal study has been officially completed globally at 48 sites across the US, Canada, Argentina, Europe, Australia and Japan. Read Roche/Genentech’s letter…

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July 15, 2019

Capricor Announces Positive Results from its Interim Analysis in the HOPE-2 Trial

Capricor Therapeutics has just announced positive results from their interim analysis in the HOPE-2 Trial after placing a voluntary dosing hold earlier this year. Please join PPMD and Capricor as we host a webinar Wednesday,…

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July 12, 2019

2019 #PPMDConference Presentations

2019 PPMD Annual Conference Resources & Recordings JUNE 27 – JUNE 29, 2019 | Orlando, Florida Thank you doesn’t begin to express our gratitude to everyone who participated in our 25th Annual Conference in Orlando,…

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July 9, 2019

NEW Resource! Large Weatherproof Duchenne Emergency Information Cards for Wheelchairs, Backpacks

In our ongoing effort to ensure that you and your family are prepared in any situation, PPMD is excited to announce the release of new large emergency cards — perfect for attaching to wheelchairs, scooters,…

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