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Duchenne Added to Minnesota’s Newborn Screening Panel
PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of Duchenne to the state’s newborn screening panel! Approximately 63,000 babies are born in Minnesota every year, which means that we expect this program to identify at least six babies with Duchenne annually once implemented. Minnesota is now the third state,…
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PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
As an early funder of PepGen, we are pleased to share that PenGen has dosed the first person in its Phase 2 trial for Duchenne amenable to skipping exon 51. Initial data , including safety and dystrophin production, at the 5 mg/kg dose is expected mid-20. Read the Press Release HERE The post PepGen Announces…
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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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Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom
AGAMREE (Vamorolone), whose development at ReveraGen Biopharma was supported by CureDuchenne, has been approved in the United Kingdom (UK) for individuals with Duchenne aged 4 years and older. The Medicines Healthcare products Regulatory Agency (MHRA) recognized not just the efficacy of AGAMREE, but also clinical benefits with regards to preserving bone health and maintaining growth…
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Introducing PPMD’s Gene Therapy Hub
We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our community continues to see progress, many new concepts and terms arise, ones that families have not had to consider before.…
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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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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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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…
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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…
