PTC Therapeutics announced the start of its fourth annual STRIVE awards program for Duchenne muscular dystrophy (DMD). STRIVE — which stands for Strategies to Realize Innovation, Vision and Empowerment — supports programs run by nonprofit patient advocacy groups that aim to increase disease diagnosis, awareness and education, and train future advocates.
The STRIVE grant awards program recognizes the vital role that advocacy groups play in giving voice to people affected by rare diseases like DMD, and intends for its program to advance their efforts supporting patients and families.
The award will allow these groups to develop unique and collaborative programs, aiming to increase the visibility of DMD, better educate both the public and healthcare professionals, improve the diagnosis and treatment of DMD, and identify and foster a new generation of Duchenne patient advocates.
“Since its inaugural year in 2015, the awards program has supported 14 patient advocacy organizations around the world that are not only committed to serving the DMD community, but pushing the boundaries in patient care by encouraging novel, innovative ways to deliver improvements in patient empowerment and information,” Mary Frances Harmon, senior vice president of corporate relations for PTC, said in a press release. “We are excited to see the novel and creative proposals that are submitted this year.”
One-year grants are awarded to qualifying organizations selected from among entries by an independent, external committee. All judges have relevant experience in rare diseases, patient advocacy and funding initiatives.
PTC’s lead therapy candidate, Translarna (ataluren), is an orally administered small-molecule compound to treat patients with genetic disorders due to a nonsense mutation, including those causing some forms of DMD.
Translarna is conditionally approved in the EU to treat nonsense mutation DMD in patients ages 5 and older who can walk. The European Medicines Agency, or EMA, has designated the drug as an orphan medicinal product, and the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for this indication. The FDA recently decided not to approve Translarna, citing inconclusive evidence regarding its effectiveness.
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