To commemorate the day, PTC will be hosting a Facebook Live event on
Bob Coughlin, President and CEO of MassBio Kelly Heger, Founder of AADC Family Network & Foundation
Irina Anselm, Director, Mitochondrial Program & Co-Director, Neurometabolic Program at Boston Children's Hospital
Matthew Klein, Chief Development Officer, PTC Therapeutics
The discussion will focus on how to raise awareness among parents and caregivers, patient advocacy groups, legislators and experts to work toward a treatment to address the high unmet medical need caused by AADC deficiency. Interested members of the public are invited to join the event via Facebook Live stream and can RSVP for the event on Facebook.
The initiative was spearheaded by
"When Jillian was diagnosed with AADC deficiency, she was one of the first children to receive the diagnosis in
"I am happy to be supporting
"We are honored to partner with other industry leaders and advocates to declare an important awareness day for the AADC deficiency community," said
AADC deficiency is a rare genetic disorder that affects the brain, causes weak muscle tone, and results in delays or failure to reach developmental milestones. Patients with severe AADC deficiency have a high risk of death during childhood and there are currently no approved therapies that address the underlying cause.2 AADC deficiency presents with signs and symptoms similar to more common diseases, and is often under and misdiagnosed by physicians, resulting in a long and challenging diagnosis journey for patients and families.
For additional information about aromatic l-amino acid decarboxylase (AADC) deficiency, please visit:
PTC is a science-driven, 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 investment in a robust and diversified 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 What is AADC Deficiency? Retrieved from https://aboutaadc.com/what-is-aadc/
2 PTC Therapeutics. (2020,