October 26, 2023

FDA Grants Approval to AGAMREE® (Vamorolone)

PPMD is excited to learn that the FDA has granted approval to AGAMREE® (vamorolone), a dissociative steroid indicated for individuals diagnosed with Duchenne muscular dystrophy from age 2 years and older.  AGAMREE® is a first-in-class…

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October 25, 2023

Duchenne Added to New York State’s Newborn Screening Panel

For more than a decade, PPMD has worked towards newborn screening (NBS) for Duchenne, including initiating a pilot study in New York State and developing a nomination package for federal review as a recommended condition…

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October 25, 2023

Community Report: Key Insights from the April 2023 Endocrine and Bone Workshop

  In April 2023, an international workshop sponsored by Parent Project Muscular Dystrophy and Duchenne Parent Project, Italy, led by Dr. Leanne Ward, Pediatric Endocrinologist at Children’s Hospital of Eastern Ontario (CHEO), gathered over 30…

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October 24, 2023

Milestone in Minnesota: Duchenne Newborn Screening Heads to Commissioner’s Desk 

For more than a decade, PPMD has been at the forefront of advocating for Duchenne newborn screening, reflecting our commitment to early diagnosis and better care. This includes the successful completion of a pioneering pilot…

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October 24, 2023

WATCH: Edgewise Therapeutics: A Novel Approach Designed to Protect Muscle in Becker (Webinar Recording)

Edgewise Therapeutics joined PPMD for a community webinar on October 18, 2023 to discuss the company’s investigational compound, EDG-5506. Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer at Edgewise, and Alan Russell, Ph.D., Edgewise’s Co-Founder and…

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October 23, 2023

Join Us for the Fall PPMD Lighthouse Workshops!

We’re excited to announce the return of PPMD Lighthouse Workshops this fall! These workshops provide a safe and supportive space for primary caregivers, as well as spouses and partners of individuals with a Becker diagnosis,…

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October 20, 2023

CDCC Spotlight: Helen DeVos Children’s Hospital

Open Doors Creating Connection At a routine multidisciplinary clinic appointment, individuals meet their neuromuscular provider, subspecialists, and ancillary staff. But what if you were able to add “meeting other kids and families” to the list…

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October 19, 2023

Sam’s Night Has Another Record-Breaking Year, Raising Over $1,400,000 for PPMD

This year’s 18th annual Sam’s Night event, hosted by the Killian Family in Dallas, TX, set yet another fundraising event record for PPMD, bringing in more than $1,400,000! Sam’s Night (formerly Sam’s Family Fun Day)…

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October 17, 2023

WATCH: Supporting Families Through Decision Making (Webinar Recording)

PPMD webinar explores how palliative care teams can support families through decision making PPMD’s Alexis Hazlett and Patrick Moeschen were recently joined by panelists Dr. Lauren Treat, Neurologist and Palliative Care Physician at Colorado Children’s,…

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October 14, 2023

WATCH: Affinity Duchenne™ and Affinity Beyond™: Updates on Investigational RGX-202 from REGENXBIO (Webinar Recording)

REGENXBIO joined PPMD for a community webinar on October 11, 2023 to discuss the company’s investigational gene therapy for Duchenne muscular dystrophy, AFFINITY DUCHENNE™, and AFFINITY BEYOND™, an AAV8 antibody assessment study. Jahannaz (Naz) Dastgir,…

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