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The things we don’t talk about as Duchenne caregivers
When three of my sons were diagnosed with Duchenne muscular dystrophy more than 10 years
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FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication
PPMD is excited to learn that the U.S. Food and Drug Administration (FDA) has accepted
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How to Identify and Manage Fat Embolism Syndrome (FES) in Fractures
Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone
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Even simple questions become complicated with FSHD
Last night, my wife, Wendy, asked me if I’d like to go out to eat.
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CureDuchenne Hosts “Champions in Miami” Event on March 9 to Help Find a Cure for Duchenne Muscular Dystrophy
DJ Irie of the Miami Heat Headlines as Event Brings Together Miami’s Top Business Leaders,
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Edgewise Therapeutics’ EDG-5506 on FDA fast track for Duchenne MD
EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation
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WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)
PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and
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Cognitive abilities stable over time in Becker MD boys, study finds
Cognitive function in boys with Becker muscular dystrophy (BMD) appear to be stable over time,
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PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development
PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea
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MDA advocacy program awards $140K to 7 rare disease groups
With projects spanning gene therapy research, patient advocacy, and air travel safety, seven U.S. organizations
