Rare Daily Staff

PTC Therapeutics said it launched Priority, an annual funding program for innovative research with a focus in its inaugural year on programs designed to improve Duchenne muscular dystrophy screening in infants and support earlier diagnosis of patients with the rare, progressive, and fatal disease.

Duchenne muscular dystrophy 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 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.

“Our hope is that each year, the Priority program will help accelerate research into innovative, practicable, and sustainable solutions to the many challenges posed by these disorders,” said Claudio Santos, senior vice president of global medical affairs for PTC Therapeutics.

The 2019 Priority program is accepting funding proposals from clinicians in the fields of neurology, genetics, and pediatrics for submission by March 31, 2019. A fund of $500,000 will be divided among successful applicants. Priority grant recipients will be announced in April 2019.

Applications will be evaluated by an external review committee comprised of independent Duchenne experts from around the world.

More information about the criteria for applications, please visit the Priority page on the PTC website.

 

February 1, 2019
Photo: Claudio Santos, senior vice president of global medical affairs for PTC Therapeutics

 

 

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