RT001 Granted Rare Pediatric Disease Designation for INAD, FA
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RT001 Granted Rare Pediatric Disease Designation for INAD, FA

In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…

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Australian Patent Issued for Trehalose (SLS-005) for Neurodegenerative Disorders
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Australian Patent Issued for Trehalose (SLS-005) for Neurodegenerative Disorders

In a press release from late January 2021, biopharmaceutical company Seelos Therapeutics, Inc. ("Seelos") announced the issuance of an Australian patent for Trehalose (SLS-005). The patent (#2019204513) centers around the…

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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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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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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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