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Friedreich’s ataxia

Neurodegenerative disease mechanism and potential drug identified – University of California – Davis

Two new studies of progressive, neurodegenerative diseases linked to defects in cells’ mitochondria offer hope for developing a new biomarker for research and…

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Discovery of a gene associated with a set of poorly understood rare diseases – Biology News Net (press release) (registration)

In this regard, IRB Barcelona is currently conducting about 20 studies in rare diseases, among them Lafora disease, Kennedy syndrome, diverse type of…

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Avalanche Biotechnologies (AAVL) and Annapurna Therapeutics Announce Merger – StreetInsider.com

… consist of Avalanche’s existing ophthalmic programs and four new gene therapy based programs, which are focused on Alpha1-antitrypsin (A1AT) deficiency, cardiomyopathy associated…

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Airdrie girls with Friedreich’s ataxia get home from anonymous donor – CBC.ca

Earlier this year, Shanna Leavitt learned both her daughters Kadence, 11, and Addison, 9, have Friedreich’s ataxia — a rare genetic neurological disease…

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Gordon Gray fundraises for Batten disease to find cure for daughters Gwenyth and Charlotte – Daily Mail

A Hollywood producer’s desperate bid to save his two young daughters from a rare brain disease has taken over Facebook and Twitter as…

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Kayaking for a cure: two men battle gale force winds to raise funds for rare disease research – ABC Online

Mr Skelton’s wife suffers from Friedreich’s ataxia, a hereditary and incurable disease affecting the central nervous system and heart. “My wife has trouble…

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