Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), in partnership with the OPTIMIZE DMD Consortium, an international consortium that aims to advance the standard of care, education, advocacy and research in endocrine and bone health for individuals with Duchenne, is proud to announce the publication of the updated version of the PJ Nicholoff Steroid Protocol for Healthcare Providers. This resource has been a lifeline for families, providing doctors with clear guidance on managing steroid dosing during illness and emergencies, and is designed to enhance the clinical management of adrenal insufficiency and steroid stress dosing for individuals on long-term corticosteroid therapies. This milestone comes as PPMD celebrates the 10th anniversary of the first publication of the PJ Nicholoff Steroid Protocol.
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The PJ Nicholoff Steroid Protocol has been instrumental in educating medical professionals on the unique care considerations for patients with Duchenne on long-term steroids who may have accompanying adrenal suppression. The newly revised edition reflects the latest advancements in clinical research, changes in the approved therapy landscape, and critical safety measures to improve patient outcomes. PPMD is grateful to those working to keep this information up to date and relative to our Duchenne community.
“At PPMD, we are committed to ensuring that every individual with Duchenne receives the highest standard of care,” said Rachel Schrader, MS, APRN, CPNP-PC, PPMD's Vice President, Clinical Care & Education. “This significant update to the PJ Nicholoff Steroid Protocol reflects the rapidly changing landscape of approved therapies and subsequent changes to clinical care. The Protocol provides expert recommendations to better equip healthcare professionals and caregivers with the most up-to-date knowledge to safely manage patients experiencing illness, injury, or safely navigating other substantive changes to their steroid regimen.”
The revised the PJ Nicholoff Steroid Protocol expands beyond the previous iteration and includes six “Critical Concepts,” including:
— Adrenal suppression of individuals on chronic steroid therapy
— Detection of adrenal insufficiency or crisis
— The need for stress dosing with expanded description of dosing and circumstances under which stress dosing is indicated
— How to safely taper when decreasing or discontinuing steroids, when indicated
— The role of stress dose steroids and return of adrenal function after tapering off steroids
— Transitioning from one steroid to another, while reducing risk of adrenal insufficiency or withdrawal
“The PJ Nicholoff Protocol is a 'how-to' guide designed to assist healthcare professionals in the prevention of adrenal insufficiency for people with Duchenne, a potentially serious complication of corticosteroid therapy,” said Leanne Ward, MD, FRCPC, a pediatric endocrinologist at the Children's Hospital of Eastern Ontario and Founder and Chair of the OPTIMIZE DMD Consortium. “The 2025 edition, developed under the expert leadership of Dr. David Weber (Children's Hospital of Philadelphia) and Dr. Anne Marie Sbrocchi (Montreal Children's Hospital), provides important updates including the management of adrenal insufficiency for individuals receiving the novel steroid, vamorolone. On behalf of the OPTIMIZE DMD Consortium, we are so grateful to the Nicholoff family for their enduring support towards international efforts aimed at the prevention of adrenal insufficiency in Duchenne muscular dystrophy.”
The updated healthcare provider-facing protocol is available for download on PPMD's website here. PPMD will be hosting a webinar on May 30, 2025, at 1:00 PM ET with leading experts to discuss its implementation in clinical practice. Register for the webinar here.
PPMD encourages families, caregivers, and healthcare providers to access this vital resource to ensure the safest care for individuals with Duchenne.
For more information or to access the PJ Nicholoff Steroid Protocol, visit parentprojectmd.org/pj.
About Parent Project Muscular Dystrophy
Duchenneis a genetic disorder that slowly robs people of their muscle strength.Parent Project Muscular Dystrophy (PPMD)fights every single battle necessary to end Duchenne.
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals.
Everything we do-and everything we have done since our founding in 1994-helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne atEndDuchenne.org. Follow PPMD onFacebook,Twitter,Instagram, and YouTube.
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SOURCE Parent Project Muscular Dystrophy (PPMD)
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