Externally Led-Patient Focused Drug Development Meeting
September 25, 2020
Access the replay here:
Voice of the Patient Report
The Voice of the Patient Report for Polyglutamine Ataxias and DRPLA is now available to download. This report summarizes information from the Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting that NAF and CureDRPLA co-hosted on September 25, 2020.
The Voice of the Patient Report for Polyglutamine Ataxias and DRPLA is a comprehensive document created from the feedback gathered at the September 25, 2020 meeting. It was submitted to the FDA. Future applications for therapy approvals will use the report as a reference when evaluating the effectiveness of the treatment.
NAF and CureDRPLA would like to offer our thanks to the patient panelists, phone callers, and individuals who submitted email comments. You bravely shared your experiences with symptoms in your daily lives. With your input, we were able to document the significant physical and emotional impact for people living with Ataxia and hopes for future treatments. We heard directly from people living with Ataxia about their struggles with coordination, walking, speech, swallowing, and tremors. This information was included in the Voice of the Patient report and will help key stakeholders understand what people living with Ataxia & DRPLA want from future treatments and clinical trials. It will ensure that the concerns of patients are taken into account.
We appreciate the efforts of the Ataxia & DRPLA community in making this a successful meeting!
Join Our DRPLA Community Today
At CureDRPLA, we connect patients, families, clinicians and researchers to further DRPLA research and work towards a treatment for DRPLA. You can join our community by going to Rare Connect. For more information about our community, get in touch with us.