Blog

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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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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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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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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PPMD: Your Strength, Your Family

PPMD: Your Strength, Your Family I had no intention of being part of the Duchenne community this long. When we started PPMD in 1994, I naively thought we would have a cure and I would…

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Join the 2019 #PPMDConference Live Stream!

Next week we will descend on Orlando, Florida for PPMD’s 25thAnnual Conference! With our most expansive agenda to date, including presentations from dozens of researchers, clinicians, and industry partners, we wanted to make this year’s…

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Sharing Our Patient Experience

PPMD has been an active voice in rare disease to ensure that our community’s experience of Duchenne is collected meaningfully and integrated fully into all aspects of the drug development life cycle. Today we participated…

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Primary & Emergency Care in Duchenne

One of the first questions parents are asked when they become pregnant is, “Who is your pediatrician?” Your pediatric primary care provider is so important that they almost a part of your family – they…

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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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Summer Camps and Immunizations for Children with Duchenne

As the school year wraps up, many families are beginning to prepare for summer activities. We have recently received questions about vaccination requirements, especially if parents are planning to send their child to camp. Specifically,…

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