Published March 13, 2018
Daniel W. Sheehan, MD, PhD, associate dean for medical curriculum and clinical professor of pediatrics, co-chaired an international panel that developed new respiratory care guidelines for patients with Duchenne muscular dystrophy (DMD).
The panel of pediatric and adult pulmonologists updated respiratory care recommendations for the 2018 Centers for Disease Control and Prevention-sponsored care considerations for the diagnosis and multidisciplinary management of DMD.
DMD is a life-shortening neuromuscular disorder that affects 15.9 per 100,000 males born live in the United States. Progressive respiratory muscle weakness and cardiac dysfunction are the major causes of death for these patients ages 20-40.
The new care considerations were published online in Lancet Neurology on Jan. 23.
Sheehan also works closely with Parent Project Muscular Dystrophy (PPMD) — a patient advocacy organization — to disseminate patient and family guides that explain these care considerations.
Sheehan says that the pace of research on DMD has accelerated, providing significant reasons to be optimistic about the future of patients dealing with the disease, which strikes boys and men in the prime of their lives.
“It is an incredibly exciting time in DMD care,” Sheehan says. “Genetic and molecular therapies for DMD are being developed at an unprecedented rate.”
For example, he noted that researchers in the Department of Physiology and Biophysics — led by Frederick Sachs, PhD, SUNY Distinguished Professor — have discovered a possible treatment based on tarantula venom. Rights to the therapy were acquired by Massachusetts-based Akashi Therapeutics, which is commercializing the drug.
Sheehan is optimistic about what the new guidelines will mean for DMD patients.
“Implementation of these 2018 multidisciplinary care considerations will greatly improve the quality of life of affected boys and men, minimizing morbidity and extending their lives,” Sheehan says. “Multidisciplinary care now can be better standardized so that the efficacy of new therapies can be fully evaluated.”
Buffalo-area patients stand to benefit from the care guidelines, as well as from new efforts on the Buffalo Niagara Medical Campus between Jacobs School faculty — who see patients at Oishei Children’s Hospital, Buffalo General Medical Center and at UBMD Pediatrics and UBMD Neurology — to strengthen multidisciplinary care.
Sheehan is starting to work with colleagues in adult and pediatric neurology to establish a multidisciplinary clinic that can apply to become a PPMD Certified Duchenne Care Center.
To receive this prestigious national certification, a center must provide patients at every visit with access to neuromuscular, pulmonary, cardiology, genetic counseling, physical therapy and social work providers, as well as a nurse care coordinator. Other specialties that should be available as needed include gastroenterology, urology, orthopedics, endocrinology, palliative care, surgery, occupational therapy, speech therapy and neuropsychology.
UBMD and Kaleida Health are well positioned to receive certification, according to Sheehan.
“With certification and standardization of care, clinical trials can then be offered on the Buffalo Niagara Medical Campus, better serving our patients and families living with DMD,” Sheehan says.