Defeat Duchenne Canada, Canada’s leading charity fighting to defeat Duchenne, and Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), have announced a collaborative research award of $300,000 (USD) in support of two Clinical Fellowships in Duchenne Endocrinology and Bone Fragility.
The three-year award will sponsor the extended fellowship of Dr. Kim Phung, as well as new fellow Dr. Rana Halloun, under the guidance of Dr. Leanne Ward, Professor of Pediatrics and Research Chair in Pediatric Bone Disorders at the University of Ottawa.
Duchenne is the most common muscular dystrophy in children, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the dystrophin gene that leads to progressive muscle weakness. A class of medications called corticosteroids (“steroids”), including prednisone, Emflaza (deflazacort), and the newly approved dissociative steroid, AGAMREE® (vamorolone), are used in Duchenne to slow down muscle damage and weakness.
Endocrine issues can be common in Duchenne, especially if a person is on steroids. When steroids are taken for an extended period, they change how the body’s natural hormones are made and work, leading to issues such as adrenal suppression, delayed puberty, and impaired growth. In addition, people with Duchenne, especially those taking steroids, have weak bones (osteoporosis), putting them at risk for fractures.
Under the guidance of Dr. Ward, the award will support the continuation of Dr. Phung’s work in clinical research while also supporting a second fellow, Dr. Rana Halloun, from The Ruth Rappaport Children’s Hospital in Israel. In 2021, Dr. Phung was awarded a two-year fellowship in pediatric endocrine and bone diseases at the University of Ottawa, with a specific focus on endocrine and bone complications of Duchenne. Both Dr. Phung and Dr. Halloun will have a specific focus on five areas, including growth and puberty, weight management, adrenal insufficiency, osteoporosis, and sexuality and fertility, among a number of other critically important initiatives pertaining to endocrinology and skeletal health care in Duchenne.
“The impact of steroids on Duchenne remains a significant area of focus for the community and our organizations, and we are pleased to partner in the awarding of these fellowships in support of Dr. Ward, Children’s Hospital of Eastern Ontario (CHEO), and the broader Duchenne community. We are eager to see the progress made under Dr. Ward’s leadership in training the next generation of Duchenne endocrinology expert clinician scientists as they tackle issues around growth, puberty, weight management, adrenal insufficiency, bone health, and sexuality and fertility.”Rachel Schrader, MS, APRN, CPNP-PC, PPMD’s Vice President of Clinical Care and Education and Lisa McCoy, Defeat Duchenne Canada CEO, joint statement