PTC: Ataluren, Clinical Trials, and Genetic Testing 101

September 29, 2021 2:00PM

Join us for a live webinar–PTC: Ataluren, Clinical Trials, and Genetic Testing 101–as part of our Community Webinar Series. Come learn about Ataluren, current clinical trials for young boys living with Duchenne (nonsense mutations), and the importance of genetic testing.

Learn more about PTC’s study to evaluate the safety and pharmacokinetics of Ataluren by reading their announcements below or joining us on September 29!

PTC’s Clinical Trial Announcement (English)

PTC’s Clinical Trial Announcement (Spanish)

Guest Speakers

Vinay Penematsa, MD
Medical Director, Clinical Development
[email protected]

Dr. Penematsa is the Medical Director and Clinical Lead for the nmDMD-Ataluren Program at PTC Therapeutics. He also supports the development of a new treatment for Mitochondrial diseases with Refractory epilepsy.

He graduated from Soochow University, in Suzhou, China in 2010. He trained at Gandhi Medical College, India where he completed his residency and practiced at Yashoda Hospitals and Little Star Children’s Hospital, Hyderabad, India.

He has interest in clinical research and committed to work towards the development of unmet medical needs in rare disease therapeutic areas. He has been involved in nmDMD clinical studies along with other indications, including Cystic Fibrosis, Huntington’s Disease, Mitochondrial Diseases with Refractory Epilepsy.


Ryan Miller, CGC
Director, Clinical Science and Diagnostics
[email protected]

As Director of Clinical Science and Diagnostics at PTC Therapeutics, Ryan works closely with biochemical and molecular laboratories to facilitate the early and accurate diagnosis of several rare genetic conditions. Prior to joining PTC last year, Ryan worked for over 5 years at Sanofi Genzyme focusing on lysosomal storage disorders. Before his move to industry, he spent 8 years working as a Genetic Counselor in a Pediatric Genetics clinic at SUNY Upstate in Syracuse, NY, seeing patients with a wide variety of genetic disorders. Ryan has an MS in Genetic Counseling from Mount Sinai School of Medicine and is a certified genetic counselor.

Community Webinar Series - Better preparing for challenges in Duchenne

Jett Foundation invites all members of the Duchenne community to attend our Community Webinar Series! This series is focused on topics tailored to help individuals and families impacted by Duchenne learn more about care, clinical trials, resources, and support. We are committed to providing these 1-hour presentations to empower individuals and families with the tools and resources needed to be their own best advocates.

View Our Upcoming Educational Webinars

Please contact [email protected] with any questions or if you’d like to become a future speaker at one of our community webinars. We look forward to having you participate in these virtual community engagement events!

Experience the magic this summer!

How is the rare disease community accomplishing goals?

Tune in on February 29 to hear from our panel and moderators on how they are accomplishing goals and living life to the fullest!

Your gift helps Jett Foundation provide programming and opportunities, such as Camp Promise, to many families impacted by Duchenne muscular dystrophy each year.

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