June 3, 2025

Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol

Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated PJ Nicholoff Steroid Protocol. Drs. Aravindhan Veerapandiyan, David Weber, and Anne Marie Sbrocchi shared valuable insights on the management of…

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May 29, 2025

PepGen Announces Discontinuation of PGN-EDO51 Development

We are disappointed to share that PepGen Inc. announced the company’s decision to end development of PGN-EDO51, its investigational therapy for the treatment of people with Duchenne amenable to exon 51 skipping. PepGen indicated that…

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May 27, 2025

Governor Abbott Signs Duchenne Newborn Screening into Law in Texas

We are pleased to share a significant milestone for the Duchenne community: Texas Governor Greg Abbott has officially signed Senate Bill 1044 into law! This legislation requires the Texas Department of State Health Services to…

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May 22, 2025

Satellos Announces Positive Functional Data from Phase 1b Open-Label Trial of SAT-3247 in Adults with Duchenne

Satellos Bioscience Inc. has announced encouraging data from the company’s Phase 1b open-label study of SAT-3247, an oral small molecule inhibitor of AAK, designed to address progressive muscle loss by restoring muscle regeneration in response…

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May 21, 2025

Florida Signs Duchenne Newborn Screening into Law

We are delighted to share exciting news for the Duchenne community: Governor Ron DeSantis has officially signed Florida’s Duchenne newborn screening legislation (HB 1089 / SB 524) into law. With this historic act, Florida becomes…

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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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