Hello friends,
As we move into summer and the country works through a phased reopening, academic and clinical researchers are coming back online with social distancing measures in place. For many academic researchers, this means lab activities beginning to resume but not at full capacity, and many lab teams working in staggered schedules to reduce the number of staff in a space at any one time.
Our Collaborative Clinical Research Network (CCRN) sites are beginning to see patients in person again for both clinical and research visits. During the first months, they will continue to work at a reduced volume and with new practices and procedures in place to protect staff and patients. We encourage you to contact your site to learn how best to proceed. Whether you are involved in ongoing research or considering participating in a new study, your coordinator can discuss options for in-person versus telemedicine visits.
Thank you for your ongoing support of FA research.
Sincerely,
Jen Farmer,
Chief Executive Officer
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IDEA Study
The IDEA research study is testing body-worn sensors to measure movement. The goal of the study is to evaluate progression and severity of ataxia. The study involves visits every 6 months over 2 years at a clinic located in Baltimore, Boston, Chicago, or Los Angeles. Participants must be 12-30 years of age and be able to walk 10-feet independently, without an assistive device. For additional eligibility criteria and clinics open for enrollment, please contact study coordinator Hannah Casey (
[email protected]
) or (773) 702-4610.
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Phase I study of CTI-1601 - Larimar Therapeutics
Recruiting FA patients ages 18 years and older with genetically confirmed Friedreich's ataxia. Must be able to go the distance of 25 feet with or without an assistive device or pushing oneself using a manual wheelchair. Click
here
for the eligibility criteria and study schedule.
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Retrotope RT001-006 Phase 2/3 trial for protection against oxidative damage in the central nervous system
Retrotope is seeking 45 volunteers to participate in a clinical trial for the drug RT001, which may protect against lipid peroxidation, the process that is believed to cause disability in many neurodegenerative diseases, including Friedreich’s ataxia. This study will involve five visits to a research clinic over a 12-month period. Sites are open at Long Beach CNS, UCLA, University of Iowa, and University of South Florida.
Click
here
for the eligibility criteria and study schedule.
FARA's Director of Patient Engagement, Susan Walther recently conducted a Q&A with Retrotope's Vice President of Medical Affairs, Dr. Mark Midei, about their study drug. You can access that interview by clicking
here
.
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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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FARA Hires Barbara Tate, Ph.D. as
New Chief Scientific Officer
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At the end of May, the FARA Board of Directors announced the hiring of Barbara Tate, Ph.D. to the full-time role of Chief Scientific Officer (CSO). Dr. Tate’s hiring marks FARA’s growing commitment of resources to FA research advancement on multiple fronts. Dr. Tate comes to FARA with a strong background in the discovery and development of therapies for central nervous system diseases. As the Chief Scientific Officer, Dr. Tate will work closely with the staff, Board of Directors and Scientific Advisory Board to set and execute FARA’s research strategy and ensure a deep and diverse treatment pipeline with well-informed and vetted treatment approaches. Click
here
to read the full press release.
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FA Research Flash Talk Series
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FARA organized the FA Research Flash Talk series that featured key aspects of FA research presented by young investigators from FARA funded laboratories around the world. Each “Flash Talk” was limited to five minutes and a single PowerPoint slide and followed by a brief Q&A session. These
presentations
are now available on YouTube -
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Chondrial Therapeutics and Zafgen Complete Merger and Begin Operating as Larimar Therapeutics
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“We are excited to complete this merger and become a publicly traded company as we develop treatments for complex rare diseases using our novel cell penetrating peptide technology platform,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics. “We believe our lead product candidate, CTI-1601, has the potential to become the first frataxin replacement therapy for patients with Friedreich’s ataxia. We are honored to have the support of both Chondrial’s and Zafgen’s existing shareholders, as well as new support from such a strong and respected syndicate of investors. We are also pleased to welcome our new directors and leadership team members, whose guidance will be instrumental as we transition to a publicly traded company and continue to advance CTI-1601’s clinical development as a potential treatment for patients with Friedreich’s ataxia, a rare disease which currently has no approved medical treatment options.”
Click
here
to read the full press release.
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The 2020 Rare Artist contest is now open for submissions! The goal with the Rare Artist Contest is to celebrate the talents of the rare disease community and spread awareness with each piece and the artists behind them. Anyone who is connected to rare is encouraged to enter, they accept entries from ages 4+.
If you are interested in participating in the Public Vote, please submit via
Facebook
or just click on the submission button below. For more details on contest
information
or
guidelines
please visit
RareArtist.org
.
If you do not have a Facebook page, or are not interested in the Public Vote, please send your submissions directly to
[email protected].
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WHEN:
August 3 to September 7, 2020
WHERE:
The local offices of your Representative and Senators (Virtual meetings may be held if in-person meetings are not possible).
WHAT:
Rare Disease Legislative Advocates (RDLA) staff organizes meetings for rare disease advocates with their Members of Congress and/or the Member's staff. The meetings take place in the Member's state or district office during the month of August, while Congress is in recess from August 3rd to September 7th, 2020. The RDLA team also helps to prepare advocates for their meetings, provides legislative resource materials, and hosts pre-meeting training webinars.
No prior experience necessary.
Registration for this event and all RDLA events are free for rare disease advocates.
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NORD Living Rare, Living Stronger
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The Living Rare, Living Stronger NORD Patient and Family Virtual Forum is only weeks away! This unforgettable weekend will bring together patients, families, medical professionals and students to learn from experts, hear inspiring stories and more!
Don't wait to register - the first 200 paid registrants will receive a goodie bag packed with a NORD zebra stress ball, a limited edition NORD zebra bandana and other surprises!
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Let Your Legacy Help End FA
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The Bartek Legacy Circle, named for FARA’s Co-Founders Ron and Raychel Bartek, celebrates donors who support the future of FARA with a gift from their estate or a life income gift. If your plans include such a gift, please let us know. We would like to thank you, make sure we understand your wishes and welcome you into the Bartek Legacy Circle. The Bartek Legacy Circle members are also gratefully recognized in FARA's Annual Report.
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Sign Up for the Patient Registry Today!
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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.
This registry is only for people diagnosed with Friedreich's ataxia.
Parents of minors diagnosed with FA can register an account on behalf of their children.
Be sure you are included in the new registry. Click the button below to enroll and to engage with resources related to living with FA.
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