Blog

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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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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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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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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PPMD Contributes to National Report on Caregiver Roles in Medical Product Development

For the last few years, PPMD has been working in partnership with the National Alliance for Caregiving, which has included the participation of PPMD’s Annie Kennedy on the planning committee of a national summit that…

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PPMD Signs on to Defense Health Research Letters

PPMD is proud to serve as a member of the Defense Health Research Consortium. Last week, letters were sent to both houses of Congress asking for their continued support for the critical and highly successful…

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PPMD Hosts Duchenne Clinical Outcomes Workshop

PPMD has been working for many years to engage critical policy makers and stakeholders within the regulatory and access environments. While we have learned a great deal over the past two years as we have…

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Newborn Screening Saves Lives Reauthorization Act Introduced in House of Representatives

Yesterday, the Newborn Screening Saves Lives Reauthorization Act was introduced in the House of Representatives. PPMD is extremely proud to be a leading coalition voice in this milestone legislation aimed at eliminating preventable newborn deaths…

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79 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget

As a result of 143 meetings with Members of Congress in Washington DC, and over 2,000 action alert emails sent by this community, we are pleased to report a very successful outcome for the Duchenne…

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Take Action before House and Senate Letters Close

Take Action before House and Senate Letters Close If you haven’t done so already please contact your Members of Congress – today – and ask them to support the FY 2020 Duchenne Appropriations Request. The…

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