The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy
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The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy

According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…

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2019 Global Genes Rare Patient Advocacy Summit Highlights: Part One

The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…

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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial
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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial

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…

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Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries

The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…

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Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?

The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…

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Phase 2 Friedreich’s Ataxia Trial Given Green Light in Spain
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Phase 2 Friedreich’s Ataxia Trial Given Green Light in Spain

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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Electrical Brain Stimulation Could Effectively Treat Neurodegenerative Ataxias

According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…

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New Expanded Access Trials For FENIB and LOTS Patients Set to Begin
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New Expanded Access Trials For FENIB and LOTS Patients Set to Begin

According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…

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