Defeat Duchenne Canada and Parent Project Muscular Dystrophy Award $300,000 (USD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility
03 Nov 2023
Drug Approval
Award Will Support Two Fellowships Under Dr. Leanne Ward, Pediatric Endocrinologist-Osteologist and Professor of Pediatrics, University of Ottawa with Focus on Endocrine and Bone Complications of Duchenne
WASHINGTON and OTTAWA, ON, Nov. 3, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), and Defeat Duchenne Canada, Canada's leading charity fighting to defeat Duchenne, today 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 specific focus on endocrine and bone complications of Duchenne. Both Dr. Phung and Dr. Halloun will have 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.
Rachel Schrader, MS, APRN, CPNP-PC, PPMD's Vice President of Clinical Care and Education and Lisa McCoy, Defeat Duchenne Canada CEO, explained in a joint statement: "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."
Dr. Leanne Ward, Research Chair in Pediatric Bone Disorders, University of Ottawa, commented, "This generous fellowship award from PPMD and Defeat Duchenne Canada represents a significant step towards advancing our understanding and management of the multifaceted challenges posed by Duchenne, particularly in the realm of endocrinology and bone health. It's a testament to the collaborative spirit of the Duchenne community and the unwavering commitment of our organizations to improve the lives of those affected by this devastating condition."
Dr. Ward recently co-chaired a workshop sponsored by PPMD titled the "Duchenne Endocrine & Osteoporosis Care in an Ever-Changing Landscape" in Ottawa, Canada, on October 26-27, 2023, a follow-up to a global workshop held in April 2023. This follow-up meeting engaged dedicated working groups as part of the Duchenne "OPTIMIZE DMD" Endocrine-Bone Consortium, ignited by the April 2023 meeting in Rome. These groups addressed various aspects of endocrine and bone health, including the impact of early steroid initiation, weight management, prevention of first fractures, growth, testosterone therapy, and addressing issues related to sexuality, fertility, and gender identity.
Additionally, Drs. Ward and Phung will co-present at Defeat Duchenne Canada's upcoming 2023 Family Forum hosted in partnership with Children's Hospital of Eastern Ontario (CHEO) on November 4th in a presentation titled "All Things Bone & Endo." During this session, Drs. Ward and Phung will delve into the latest developments and crucial topics related to bone health, growth, puberty, and adrenal insufficiency for Duchenne muscular dystrophy.
ABOUT DEFEAT DUCHENNE CANADA
One in every 5,000 boys is born with Duchenne muscular dystrophy, the most common fatal form of muscular dystrophy.
The disease is relentless. It slowly weakens the body's muscles, deteriorating function of vital organs and ultimately - shortens their life. Although there are medical treatments that may help slow its progression, there is currently no cure.
Defeat Duchenne Canada is the country's only national charity dedicated to ending Duchenne muscular dystrophy. We have provided leadership in research, advocacy, and support since 1995. We'll continue until a cure is found to ensure our boys can live long and active lives.
Join our fight to defeat Duchenne at defeatduchenne.ca. Follow Defeat Duchenne Canada Facebook, Twitter, Instagram, LinkedIn and YouTube.
ABOUT PARENT PROJECT MUSCULAR DYSTROPHY
Duchenne is a fatal 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 seven 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 at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.
SOURCE Parent Project Muscular Dystrophy (PPMD)
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