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Coping with seasonal affective disorder and FSHD as best I can
The symptoms of my facioscapulohumeral muscular dystrophy (FSHD) make staying positive a daily challenge. Many people with muscular dystrophy struggle with depression. This battle is magnified every winter as I also feel the impact of seasonal affective disorder (SAD). Exposure to sunlight helps our bodies produce vitamin D, a compound that can help boost serotonin…
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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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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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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…
