May 12, 2025

Budget Reconciliation Update and the Impact on Medicaid

PPMD is closely monitoring the U.S. House of Representatives Energy and Commerce Committee markup of the Committee’s budget reconciliation text, scheduled for tomorrow, Tuesday, May 13, 2025, at 2:00 PM ET. Updated legislative language released…

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May 12, 2025

A Decade of Impact: Honoring PJ’s Protocol and Its Legacy

Ten years ago, the Duchenne community gained a vital tool for the clinical management of adrenal insufficiency and steroid stress dosing for individuals on long-term corticosteroid therapies: the PJ Nicholoff Steroid Protocol for Healthcare Providers….

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May 7, 2025

An Update on PPMD’s Continued Strategy for Advancing Duchenne Newborn Screening

On April 3, 2025, the U.S. Department of Health and Human Services (HHS) announced the dissolution of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), an unexpected development that has left a…

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May 1, 2025

Share your NOW: The impact of up-to-date data in The Duchenne Registry

The Duchenne Registry is designed as a longitudinal (long-term) research study, collecting information from the same people about the same topics over time. Data entered at one point in time is just that – a…

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April 28, 2025

Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati Children’s Hospital, and Dr. Craig…

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April 28, 2025

The BELS Questionnaire: A New Tool to Support Your Child’s Emotional Wellbeing

PPMD is excited to share that a new tool, the BELS (Behavioral, Emotional, Learning, and Social) questionnaire, has been developed by Drs. Natalie Truba (Nationwide Children’s Hospital) and Molly Colvin (Massachusetts General Hospital) and was…

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April 21, 2025

Watch: Duchenne Newborn Screening – Next Steps Following ACHDNC Dissolution (Webinar Recording)

Last week, PPMD and the Muscular Dystrophy Association provided insights into the current status of the Duchenne nomination to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening following the dissolution of the Advisory…

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April 11, 2025

Congress Moves Forward on Medicaid Cuts: What You Need to Know

This week, we received concerning news from Washington: Congress has passed a budget resolution that will require significant cuts to Medicaid—up to $880 billion over 10 years. Congress is still determining the exact scope and…

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April 8, 2025

Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with…

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April 4, 2025

The Future of Newborn Screening for Duchenne: Moving Forward After the ACHDNC’s Dissolution

Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has been terminated, effective immediately. For over 20 years, this committee has served as the guiding body for adding…

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