Parent Project Muscular Dystrophy

Collaborative definition of patient centricity by AstraZeneca, patients and carers

Friday, March 31, 2017

AstraZeneca has announced that BMJ Innovations has published the first collaborative definition of patient centricity for the pharmaceutical industry. The definition was co-developed with patients and caregivers using research led by AstraZeneca and patient community advocates to ensure a deep understanding of patient needs and values. Ten associated patient-centric principles were also identified and published in BMJ Innovations to provide the pharmaceutical industry with a consistent reference point to sustain meaningful and mutually beneficial engagement with patients. Wider testing of these outputs among U.S. and European patient communities confirmed their validity.

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PTC Therapeutics cuts workforce 18%

Thursday, March 24, 2016

PTC Therapeutics is reducing its workforce by approximately 18%, which will primarily affect employees and contractors in the U.S. This reduction is part of PTC’s program intended to optimally manage operating expenses following its recent setback related to the Refuse to File letter received from the FDA for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). 

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PPMD calls for greater transparency, accountability of FDA patient engagement efforts

Wednesday, December 10, 2014

Parent Project Muscular Dystrophy (PPMD), an advocacy organization working to end Duchenne muscular dystrophy, is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process. PPMD is advocating for provisions within the 21st Century Cures Initiatives focused on measuring how the agency is or is not using new patient engagement tools.

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