Potential Treatment for Friedreich’s Ataxia Earns Orphan Drug and Rare Pediatric Disease Designations
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Potential Treatment for Friedreich’s Ataxia Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…

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New Hope for ALS and Friedreich’s Ataxia Patients Through Viral Vector Transports
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New Hope for ALS and Friedreich’s Ataxia Patients Through Viral Vector Transports

A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…

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RT001 Granted Rare Pediatric Disease Designation for INAD, FA
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RT001 Granted Rare Pediatric Disease Designation for INAD, FA

In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…

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How Children With Disabilities Are Excluded in Schooling During the COVID-19 Pandemic
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How Children With Disabilities Are Excluded in Schooling During the COVID-19 Pandemic

An Australian royal commission is investigating claims that children with disabilities are being excluded from online learning, according to The Guardian. They are also looking into issues with the services…

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Patients Recruited for Phase 2 Study of Experimental Friedreich’s Ataxia Treatment

According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich's ataxia treatment, MIN-102 (generic…

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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial
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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial

According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…

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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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I Have a Rare Disease And I Am Crossing Borders
23/02/2016 Pics (C) Huw John, Cardiff. MANDATORY BYLINE - Huw John, Cardiff Alan Thomas from Llanboidy HealthWise Wales, a new project which will inform future generations’ NHS services, to help protect against fatal illnesses. e-mail: mail@huwjohn.com Web: www.huwjohn.com

I Have a Rare Disease And I Am Crossing Borders

My name is Alan. I cross many borders as a person with a rare disease. These borders are physical, geographical and psychological. Crossing so many borders has earned me the…

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Editor’s Choice: Rare Adolescence, Rare Parenting and Rare… CEOing

Happy Memorial Day Weekend Patient Worthians! This week we have an inspiring interview with the CEO and Founder of the Hereditary Neuropathy Foundation. We also have a motivating story on a cheerleader battling Friedreich's…

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This Mother’s Amazing #showusyourshoes Campaign for Friedreich’s Ataxia
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This Mother’s Amazing #showusyourshoes Campaign for Friedreich’s Ataxia

Leah Alstin has a rare disease called Friedreich’s ataxia. Friedreich’s ataxia is a disease that progressively damages and affects the nervous system. Poor coordination is one of the early symptoms…

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High School Senior With Friedreich’s Ataxia Fulfills Cheerleading Dream
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High School Senior With Friedreich’s Ataxia Fulfills Cheerleading Dream

Kiara Trussell thought her dreams of being a cheerleader would never happen. This is partly because Friedreich’s ataxia has made her wheelchair bound. Friedreich’s ataxia is a progressive neurological condition.…

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Parenting a Child with Friedrich’s Ataxia? Here’s a Resource for You
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Parenting a Child with Friedrich’s Ataxia? Here’s a Resource for You

Having a difficult time finding resources on Friedreich's ataxia (FRDA)? Even for the generation that grew up with Google, keeping up with the news online can be tricky. You’ve got…

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How is Friedreich’s Ataxia Linked to Inflammatory Bowel Disease and Growth Hormone Deficiency?
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How is Friedreich’s Ataxia Linked to Inflammatory Bowel Disease and Growth Hormone Deficiency?

Friedreich's ataxia (FA) is a rare and painful disease. With few treatments available to treat the genetic mutation, most therapy is aimed at controlling symptoms. Clearly, these short-term band-aids aren't…

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