Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
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…
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia. Friedreich’s ataxia (also known as…
Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
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For a long time there have been no treatments for Friedreich's ataxia. With no FDA approved therapies, people with this disease have only been receiving symptomatic treatment. Exicure has changed…
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