Friedreich’s ataxia

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say – Friedreich’s Ataxia News

Denise Cloutier, lab manager at the University of Florida’s Powell Gene Therapy…

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Woman gets mistaken for her fiance’s CARER due to his disability – Daily Mail

A woman who gets mistaken for her fiancé’s carer has revealed how their relationship is…

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Another myostatin program combusts, but Acceleron has plenty in the pipeline – Endpoints News

Myostatin inhibitors — engineered to build muscle and improve mobility in patients — theoretically make…

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FDA revamps adverse event reporting guidance for Covid-19 – Endpoints News

  The FDA on Thursday revised its guidance…

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Ryanair refuses to carry disabled woman’s wheelchair on flight – The Independent

A disabled woman was told she could not take…

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Glenn McCrory: That Night I Fought for the Title, David Was Pulling Me Along – Hannibal Boxing

Glenn “Gentleman” McCrory celebrates after defeating Patrick “Killer” Lumumba by unanimous decision…

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Mum with rare condition mistakenly stopped for ‘drink driving’ and refused nightclub entry – Derbyshire Live

A Belper mum-of-two who was refused nightclub entry and stopped by the police due to misunderstandings surrounding…

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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…

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Discovery of a gene associated with a set of poorly understood rare diseases – Biology News Net

The genomic sequencing of hundreds of patients with diverse…

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

Avalanche Biotechnologies, Inc. (Nasdaq: AAVL) and Annapurna Therapeutics SAS,…

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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…

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

Rare genetic disease becomes a massive celebrity cause after…

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

While many Australians enjoyed a leisurely Labour Day long…

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