Dear friends,
The Pathway donors help fund research progress through a monthly gift to FARA. Since the program was launched in August 2020, this community of donors has raised over $110,000 for FA research! This amount is equivalent to the support needed to fund an investigator at an FA research lab bench for two years. We invite you to join The Pathway campaign and donate $10 a month or more towards FA research. Our aim is to grow the program by 25 supporters this month. Click here to learn more information and join The Pathway.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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Reata Pharmaceuticals Announced FDA Filing Acceptance and Priority Review Designation for the New Drug Application (NDA) for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia.
The NDA is supported by the efficacy and safety data from the MOXIe Part 2 trial and additional supporting data from the MOXIe Part 1 and MOXIe Extension trials.
The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis, or prevention of a serious condition. The FDA has assigned a Prescription Drug User Fee Act (“PDUFA”) target action date of November 30, 2022. The FDA indicated it is currently planning to hold an advisory committee meeting to discuss the application.
Read the full press release here.
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The FARA Grant Program is pleased to announce that the Award for Innovative Mindset has been granted to Ankur Jain, PhD, Whitehead Institute for Biomedical Research, & Ricardo Mouro Pinto, PhD, Harvard Medical School and Massachusetts General Hospital for a new ultrasensitive single-molecule assay for frataxin measurement.
These investigators propose to develop a sensitive and quantitative assay for measuring frataxin levels that could help drug development in FA as well as advance our understanding of disease mechanism. Direct measurement of frataxin remains challenging owing to its low abundance and the inherent detection noise of commonly used assays. Dr Jain and Dr Mouro Pinto propose to overcome this hurdle by implementing a technology that allows detection of very small amounts of molecules directly from serum, blood, or tissue extracts. This method, called SiMPull, combines immuno pull-down (isolating a molecule with antibodies) with single-molecule microscopy, allowing to visualize single protein molecules with a microscope. This assay was recently utilized to measure a variety of biomarkers in blood present at very low concentrations. The investigators hypothesize that the increased sensitivity, quantitative readout, and dynamic range afforded by SiMPull will allow them to accurately quantify frataxin levels, including measurements of subtle changes that are undetectable by conventional methods. SiMPull may provide opportunities to use frataxin in accessible biospecimen such as blood, serum, and cerebrospinal fluid as a biomarker for disease progression and for assessing the effectiveness of therapies in clinical trials.
The FARA Grant Program awards competitive funding for research that advances the understanding of Friedreich Ataxia. Learn more here.
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Tune in to the 5th annual Cure FA Soirée on June 25th at 6:30 pm central! Watch the live stream at live.curefasoiree.org. The Soirée is an evening of the arts for all ages, and all proceeds go to FARA. Make a donation at give.curefasoiree.org. Last year‘s Soirée (FAer pic below) raised $155K; this year‘s goal is $175K! Learn more at curefasoiree.org.
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Ever wonder about the impact of your support on FA research? FARA, in collaboration with members of the FA community, created a series of short, animated videos to help explain how your research dollars are put to work. The first of these videos can be found here. Look for a new episode every month.
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Congratulations, Michael Crawley!
2022 Myra Kraft Community MVP Award Recipient
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Congratulations to Race for Matt & Grace Event Chair and FARA Finance Committee member, Michael Crawley for being selected as one of the recipients of the 2022 Myra Kraft Community MVP Awards. This award celebrates volunteerism and those who have gone above and beyond in their communities. Thank you to the New England Patriots Foundation for honoring Michael and supporting FARA!
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Save the Date - September 24- 25, 2022!
FA Symposium
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The Friedreich's Ataxia Research Alliance (FARA) and the Friedreich’s Ataxia Center of Excellence at Children’s Hospital of Philadelphia (CHOP) are pleased to present this hybrid in-person and virtual symposium providing patients and families with up-to-date information about current research and therapeutic approaches in Friedreich’s ataxia.
This symposium will be held on September 24th & 25th 2022 at the Sheraton Valley Forge in King of Prussia, PA. The symposium will begin with a welcome reception Saturday, September 24th from 5:00pm - 6:30pm (ET). The general session and presentations will follow on Sunday, September 25th from 8:00am - 4:30pm (ET).
Click below to register and select in-person or virtual participation.
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Researchers at Vanderbilt University Medical Center in Nashville, TN want to learn more about changes in glucose control and cardiac features in children with Friedreich's Ataxia. They are recruiting participants (ages 7-17 years) with FA for a study to determine if there is a relationship between blood sugar abnormalities (risk of diabetes) and cardiac changes.
If your child is selected to participate in this study, your child may complete blood testing, oral glucose tolerance testing (with a non-FDA approved stable isotope product), placement of a continuous glucose monitor, echocardiogram (ultrasound of the heart), and dual-energy X-Ray absorptiometry (DXA, x-ray scan) during a 1-2-day study visit. You and your child will be compensated for your time. Click here for more information about this study.
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This is a first-in-human, Phase 1 study of DT-216 which means that trial will begin with single dose cohorts to assess safety. Enrollment will be in cohorts (typically 5-8 people). The first cohort will be enrolled and tested at the first dose, then there will be a review of the safety, then typically several weeks later the next cohort will be enrolled to test the next dose. After the single dose study, there will be a multiple dose study, which is also a safety study. Multiple dose studies typically assess safety over a period of several weeks to a month. More information on the multiple dose study will be available once the single dose study is further along.
Inclusion criteria:
- Individuals with FA ages 18 to 55 years old (inclusive) at screening
- Genetically confirmed diagnosis of FA with homozygous GAA repeat expansions
- Weight between 90 and 200lbs (approximately)
- Ability to sit upright with thighs together and arms crossed without requiring support on more than two sides
- Ability to perform basic daily care such as feeding yourself and basic personal hygiene with minimal assistance
- Must have completed full COVID-19 vaccination at least 4 weeks before treatment
This study is being conducted at Clinilabs and will require about a week long stay at the study site. Compensation will be provided for qualified participants and caregivers. Individuals and caregivers who are not local to the research facility will receive no-cost transportation and accommodations. Click here for more information and to read the study flyer.
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Please consider enrolling in this important study. Participants needed at all study sites.
TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome.
Eligibility criteria include:
- Age >5 years with genetically confirmed diagnosis of FA.
- GAA repeat expansion >55 in length on both alleles of the FXN gene or GAA repeat expansion on one allele and another mutation type on the other allele (e.g., point mutation, gene deletion).
- Age of disease onset less than 25 years.
- Disease duration less than 25 years.
- Able to undergo MRI
- Participants can be involved in an investigational drug trial and still be eligible for the TRACK-FA study.
There are 3 study visits, each approximately 12 months apart. At each study visit, participants will be asked to complete neurologic and functional testing, blood draw and brain and spinal cord MRI scan.
Sites are open for enrollment at University of Minnesota (Minneapolis), University of Florida (Gainesville), Children’s Hospital of Philadelphia (Pennsylvania), RWTH Aachen University (Aachen, Germany), University of Campinas (Brazil), and Monash University (Victoria, Australia). To contact a study coordinator, please click here.
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If you are selected to participate in this study, you would complete 2 in-person study visits, each lasting 2 days, over a 12-week study period at CHOP and Penn. During the study period, you may complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.
Participants will be compensated for time in the study, and participants will be provided with a recumbent bike, which they can keep after study completion.
To learn more about the study and determine your eligibility, contact the study coordinator, Anna DeDio, by email dedioa@email.chop.edu or by phone
(267) 425-1998.
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Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular study. |
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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.
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