OPTIMIZE DMD

PJ NICHOLOFF STEROID PROTOCOL

Individuals with Duchenne Muscular Dystrophy (DMD) who take glucocorticoids (including prednisone, prednisolone, deflazacort, and vamorolone) are at risk of adrenal suppression. This means their bodies cannot produce cortisol normally during times of stress. All patients on steroids need a stress steroid plan for illness, injury, or surgery and must follow a careful tapering schedule when reducing or stopping steroids to prevent potentially life-threatening complications.

PJ NICHOLOFF STEROID PROTOCOL applies to individuals with muscular dystrophy who are treated with glucocorticoids (GCs), including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy, female manifesting carriers, and X-linked cardiomyopathy (collectively abbreviated as DBMD). 

Vamorolone (AGAMREE®) Information Sheet for Healthcare Providers

Vamorolone (AGAMREE®), is a new steroid used to treat Duchenne muscular dystrophy (DMD). It is designed to offer the same muscle-strengthening benefits as classic steroids—such as prednisone, prednisolone, and deflazacort—while potentially reducing some of the common side effects associated with these medications.

This information sheet has been prepared by OPTIMIZE DMD to provide necessary information for Healthcare Providers to help individuals living with DMD and their families better understand the role of Vamorolone in DMD treatment.

A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing Landscape

In April 2023, over 30 experts and advocates from four countries met in Rome, Italy to discuss unmet needs in endocrine and bone health care for individuals with Duchenne muscular dystrophy (DMD). Despite recent advances in muscle-targeted therapy, long-term glucocorticoids (GC) remain the backbone of treatment for the foreseeable future. This affirms the need to intensify efforts that will mitigate serious complications of GC therapy, including unexpected mortality due to fat embolism syndrome following bone injury and also unrecognized adrenal suppression, early loss of ambulation linked to excess weight and/or fragility fracture, adverse cardiometabolic effects of GC, the psychosocial impact of profound growth and pubertal delay/hypogonadism, and the burden to families arising from monitoring and treating endocrine and skeletal complications of GC therapy. Delegates discussed: 1. The impact of GC therapy on the heart, 2. Predictors of fragility fractures and experience with intravenous and oral bisphosphonates plus teriparatide, 3. The effect of hormonal therapy on muscle-bone health, 4. Adrenal suppression, 5. Weight management, 6. Puberty, sexuality, fertility and gender identity, 7. The impact of early GC initiation, 8. Emerging knowledge about Vamorolone (a novel dissociative steroid) and its effects on muscle, bone and endocrine health, and 9. Experiences implementing an endocrine-bone health management strategy nation-wide (in the UK). At the conclusion of the meeting, it was agreed that an endocrine-bone working group should be struck to continue the narrative, following which the International OPTIMIZE DMD Consortium was ignited to move the dial in these important areas.