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Join us at PPMD’s 30th year meetings and events
Happy New Year! As PPMD celebrates its 30th anniversary in 2024, we’re thrilled to announce the expansion of our events and meetings dedicated to accelerating research, enhancing care and quality of life, and fostering connections within our amazing Duchenne and Becker family. From launching our new PPMD Together regional meeting series for families to connect…
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Juvena’s JUV-161 named FDA orphan drug for DM1
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead treatment candidate for myotonic dystrophy type 1 (DM1). Orphan drug status is designed to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides benefits such as seven years of market…
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PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024
Join PTC Therapeutics and CureDuchenne for this prerecorded webinar to hear important information regarding Emflaza, PTC Cares and the programs that support the Duchenne community. Topics include:Understanding Your PrescriptionHow to Ensure Your Son Has Access to EMFLAZAWhat does Dispense as Written (DAW) mean? How do DAW products and generics differ in terms of patient support?Why would I receive a generic?and How can I make sure…
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Phase 3 trial of SRP-9003 gene therapy in LGMD2E starts screening
Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E (LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without the ability to walk independently. It will first include a…
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CureDuchenne Welcomes Patricia Brown as Senior Director of Community Engagement
Seasoned Healthcare Professional Brings Experience in Patient Advocacy and Community Education to Global Rare Disease Nonprofit Newport Beach, Calif. – January 22, 2024 – CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, today announced an addition to its executive team with the hiring of Senior Director of Community Engagement,…
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CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy
Upcoming Events Across the Country Provide Latest Treatment Information and Resources for Managing Challenges of Living with Duchenne or Becker and Improving Quality of Life NEWPORT BEACH, Calif., January 22, 2024 – CureDuchenne, a global leader in research, patient care and innovation for improving and extending the lives of those living with Duchenne muscular dystrophy, has…
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Caregiving for sons with DMD is like being a frog in boiling water
One of my favorite early-summer memories is from when my sons Max and Rowen were younger. Both lovely little boys — one blond-haired and blue-eyed, the other dark-haired and brown-eyed — squealed with laughter upon discovering a frog in our front yard. Max held the frog up in his hands and away from our yellow…
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Next-gen gene therapy SGT-003 named FDA orphan drug
SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined as conditions affecting fewer than 200,000 people…
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Astellas Releases Results of Discontinued Phase 1b Study of ASP0367
Astellas has shared results of the Phase 1b study of ASP0367 (MA-0211), an oral investigational drug. The study aimed to evaluate safety, tolerability, and preliminary efficacy in individuals living with Duchenne aged 8 to 16 years old. The study was stopped early, in November 2022, because not enough patients were available to take part in the study. While…
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PPMD Hosts 2024 Duchenne Healthcare Professionals Summit
Each January, PPMD brings together experts in the field for our Duchenne Healthcare Professionals Summit to ensure that caring for individuals with the latest information and technology remains a constant priority. Healthcare professionals from PPMD’s Certified Duchenne Care Centers and other neuromuscular programs providing dystrophinopathy care, as well as researchers, academics, and industry partners, gather…
