Dear friends,
In FARA's strategic plan, one of the core pillars is to share knowledge and know-how within the research and patient/caregiver communities. Some of the resources previously developed by FARA to help educate the FA community about clinical research include a series of self-paced Gene Therapy Learning Modules as well as a Clinical Trials Guide for Participants.
To add to this growing library of education materials, FARA recently created a Rare Disease Medication Frequently Asked Questions (FAQ) document. As programs advance through the Friedreich's Ataxia Treatment Pipeline, FARA has been researching how other rare disease patients access their approved medications. During this research, we learned some new vocabulary along with processes and programs. We outline some of our learnings in this FAQs document. We hope you find it useful. As we continue to identify education opportunities and resources, we are committed to sharing that information with the FA community.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
|
|
Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia
Design Therapeutics, Inc., a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, reported progress across its portfolio of novel GeneTAC™ small molecules. This update includes the initial results on DT-216 from the company’s single-ascending dose (SAD) Phase 1 clinical trial in patients with Friedreich ataxia (FA). The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response. These data support the continued advancement of DT-216 in the ongoing multiple-ascending dose (MAD) Phase 1 trial and the anticipated Phase 2 clinical trial in FA patients, which is on track to begin in 2023.
Click here to read the full press release and more about the study.
|
|
There are several clinical studies and trials open and enrolling participants. Click the titles below to learn more about the research studies, participating sites, and contact information for study coordinators.
|
|
Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.
|
|
|
Check out the final 2022 episode of the Minutes of Science series to learn more about your impact on FA research this year! You helped FARA fund a global grant program, lead large-scale collaborative research projects, develop clinical research infrastructure for trials, and co-host the largest ataxia scientific conference to date. Thank you for your participation and support!
|
|
Recently Funded FARA Grants
|
|
- The Keith Michael Andrus Cardiac Award has been granted to Laura Kropp, PhD, MPH at Stealth Biotherapeutics to Advance Novel Mitochondrial Therapies for FA Cardiomyopathy.
- An Outcomes & Biomarkers General Research Grant was awarded to Louise Corben, PhD, Murdoch Children's Research Institute (MCRI), to measure ataxia in children with Friedreich Ataxia.
- A two-year General Research Grant was awarded to David Lynch, MD, PhD, William Gaetz, PhD & Tim Roberts, PhD from the Children’s Hospital of Philadelphia for Imaging of glutathione and GABA in the brain as biomarkers of Friedreich Ataxia.
-
Pei-Yu Chen, PhD, at Yale University, was the recipient an Award for Innovative Mindset to study TGFβ signaling activity as disease biomarkers and therapeutic targets in Friedreich’s Ataxia.
- Joseph Nabhan, PhD, from Vesigen Therapeutics, was awarded a General Research Grant for the evaluation of ARMMs-mediated delivery of Cas9 protein complexed with gRNAs as a non-viral disease-modifying strategy for Friedreich’s ataxia.
Learn more about these awards and the grant program here.
|
|
Thank you to all of our advocates who helped get Friedreich’s ataxia on the list of eligible conditions for the Congressionally Directed Medical Research Program (CDMRP) in 2022. This allowed FA researchers to apply for grant funding, and six FA grants were recommended for CDMRP/PRMRP FY22 funding totaling over $14 Million!
Click here to subscribe to FARA’s Advocacy Newsletter and receive updates on ways to be involved in advocacy efforts throughout the year.
|
|
Save the Dates - rideATAXIA 2023
|
|
Save the Dates for the rideATAXIA 2023. Hope to see you at one or all of these rides!
- rideATAXIA Gainesville - March 26th
- rideATAXIA Chicago - July 9th
- rideATAXIA Philadelphia -October 1st
- rideATAXIA Dallas - November 4th
And rideATAXIA teammates in California and beyond, stay tuned for the launch of the new rideATAXIA Hometown program. rideataxia.org
|
|
The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed.
|
|
|
|
|
|
|