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Design Therapeutics Reports GeneTAC™ Portfolio Progress and

Design Therapeutics Reports GeneTAC™ Portfolio Progress and
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One-in-lifetime opportunity : Huge fundraiser to help Old Colwyn dad travel to Serbia in bid to restore sight

A HUGE fundraiser to raise £24,000 has been launched to enable a father-of-two to travel to Serbia in an attempt to get his sight back. Ben Francis, 25, was diagnosed with a disease called Friedreich Ataxia, a rare inherited disease that causes progressive nervous system damage and movement problems, at the age of 11. Ben, of Old Colwyn, who has partner Sara Ormond, struggles to do simple tasks such as cooking, pouring a glass of milk and getting around his home, but his biggest challenge is the loss of his eyesight. Ben is not completely blind, he can see shapes and movement, but time is running out as Ben is deteriorating fast.

One-in-lifetime opportunity : Huge fundraiser to help dad travel to Serbia in bid to restore sight

Friedreich s Ataxia Market Analysis, Leading Companies, Emerging Drugs, and Epidemiology Forecast

(MENAFN - GetNews) DelveInsight s Friedreich s Ataxia Market Insights, Epidemiology, and Market Forecast 2030 report delivers an in-depth understanding of the Friedreich s Ataxia, historical and forecasted epidemiology as well as the Friedreich s Ataxia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. Friedreich s Ataxia (FA) is a rare autosomal recessive neurodegenerative disease that causes progressive nervous system damage and movement problems. It usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time. Most patients carry homozygous GAA expansions in the first intron of the frataxin gene on chromosome 9. Frataxin is a mitochondrial protein. Due to epigenetic alterations, frataxin expression is reduced significantly, and its deficiency leads to mitochondrial iron overload, defective energy supply, and generation of reactive oxygen species.

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