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…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
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…
Sometimes, medical studies just don't go the way that companies want them to. According to MedCity News, this is what biotechnology company Larimar Therapeutics ("Larimar") was faced with during…
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…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
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…
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…
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…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
Many times, diseases can take very physical tolls on a person’s body. These physical symptoms can sometimes be uncomfortable, they can be painful, and sometimes they may even be embarrassing…
According to a story from Rare Revolution Magazine, Georgia Hart was first diagnosed with Friedrich's ataxia when she was 15 years old. Since then, living with this rare disease has…
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…
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…
Anna Gordon from West Virginia doesn't let her Friedreich ataxia define her, and she encourages anyone with a rare disease to not let it define them either, reported Public Broadcasting.…
When Raeno Brendtro was asked to sit down after her diagnosis, she refused. She needed to stand for as long as she could, now that her time was limited. The…
When you have a rare disease like Friedreich ataxia (FA), which affects only 50,000 people in the United States, it is pretty easy to feel alone and isolated. Usually, the…
If you or someone you love have Friedreich's Ataxia, you know how badly we need an adequate treatment. Friedreich's Ataxia (FA) is a rare disorder, which results in neurological and…
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…
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…
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.…
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…
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…
Whenever we’re struggling with something, we assume we are alone. We feel like we are the only ones. But in September of 1997, a small group of Friedreich’s ataxia (FA)…
Before we take a look at a rare disease called Freidrich ataxia, or FA, let's first understand the meaning of the word "ataxia." Ataxia includes loss or lack of muscle…
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