"As a research-driven, patient-centric company, it's important to PTC that we support the efforts of clinicians and researchers who share our passion for improving the clinical care of people with rare, genetic disorders," said Dr.
The 2019 PRIORITY program invites funding proposals from clinicians in the fields of neurology, genetics, and pediatrics for submission between now and
Funding applications will be evaluated by an external Review Committee comprised of independent Duchenne experts from around the world.
"This year's program will recognize innovative initiatives to improve screening for Duchenne in infants that will ultimately lead to a much earlier diagnosis of this progressive disease," said Dr.
Duchenne muscular dystrophy is a rare disease and affects approximately 1 in every 3,600–6,000 live male births.1 Despite improvements in clinical care over the last 20 years, there remains a delay in the diagnosis of children with Duchenne (mean age of diagnosis averaging between 4–4.5 years, with many diagnoses occurring much later). This delay reduces the benefits of post-diagnosis interventions that can delay loss of ambulation and extend quality of life.2,3,4,5
For more information around the criteria for applications, please visit the PRIORITY page on the PTC website: www.ptcbio.com/priority
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information on the signs and symptoms of Duchenne can be found at: www.duchenneandyou.com
About PTC Therapeutics, Inc.
PTC is a science-led global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives our continued investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
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1. Bushby K et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurology. 2010;9(1):77-93.
2. Van Ruiten HJ et al. State-Of-The-Art Advances in Muscular Dystrophy.
3. Ryder S et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review.
4. Van Ruiten HJ et al. Improving recognition of Duchenne muscular dystrophy: a retrospective case note review. Archives of Disease in Childhood. 2014;99(12):1074–7.
5. Ciafaloni E et al. Delayed diagnosis in Duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MDSTARmet).