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 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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June 7, 2019

PTC Receives FDA Approval for Expansion of EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age

PTC Therapeutics announced that they have received FDA approval for the expansion of the EMFLAZA® (deflazacort) labeling to include patients 2-5 years of age. Emflaza was first approved by the FDA in February 2017 for…

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May 31, 2019

WATCH: Domagrozumab as a Potential Treatment for Duchenne – Webinar Recording

On May 22, 2019, Pfizer Inc. joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of the primary safety and efficacy results from the Phase 2 clinical trial. Additional…

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May 20, 2019

PPMD’s Certified Duchenne Care Center Program – 2018 Impact & Progress

For 25 years, care has been at the heart of everything PPMD does. We strive to ensure that people living with Duchenne are living longer, stronger lives, by helping you access expert healthcare providers, a…

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May 14, 2019

Solid Biosciences Provides Update on IGNITE DMD Status and New Clinical Sites

Solid Biosciences shared an update on the progress of their IGNITE DMD Phase I/II clinical trial for investigational microdystrophin gene transfer, SGT-001. The update includes news about initiating dosing for their higher dose cohort of…

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