NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
CENTRAL TIME ZONE
In this session, we will learn what gene therapy is, the different types of gene therapy, and how gene therapy could be used to treat Ataxia in the future. We will also discuss the limitations of gene therapy technology, as well as what research is being done right now on gene therapy for Ataxia. If you have questions about gene therapy, this webinar is for you.
Register: Click here.
Carolyn Yrigollen, PhD
Children’s Hospital of Philadelphia
Dr. Yrigollen is a researcher at the Children’s Hospital of Philadelphia with 10 years of experience working on gene editing systems to treat heritable neurological disorders. Carolyn earned her Ph.D. in genetics from the University of California, Davis where she studied the structure and function of the Fragile X gene. She is currently working on CRISPR based approaches to correct the underlying mutations leading to Fragile X Syndrome and Fragile X-associated Tremor/Ataxia Syndrome.
NAF hosts educational webinars about Ataxia presented by industry experts. Learn about important topics related to living with Ataxia. Webinars are complimentary for NAF members, with new sessions offered each month. Join NAF to receive invitations to upcoming Ataxia webinars. Visit www.ataxia.org/webinars to explore past webinars on a variety of topics.
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