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Ataxia

As Potential Therapy for Friedreich’s Ataxia, Researchers Study RNA Transcript Therapy to Deliver Frataxin mRNA – Friedreich’s Ataxia News

Friedreich’s ataxia is an autosomal recessive inherited disease that causes progressive damage to the nervous system. Although it is primarily a neurodegenerative disease…

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Friedreich’s Ataxia Clinical Study of Oral Drug, RT001, Enrolling … – Friedreich’s Ataxia News

Here, Retrotope announces start of patient enrollment for a high-dose cohort of RT001, a potential Friedreich’s ataxia treatment, now in a Phase 1-2…

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DHA & The Boom Room Present: Ataxia – Deep House Amsterdam (press release) (blog)

Alongside their activities in the studio, Ataxia perform at some of North America’s best clubs, such as Standard Rooftop in Downtown LA, Audio…

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Orphan Drug Status Granted to BHV-0223 for Spinocerebellar Ataxia – Monthly Prescribing Reference (registration)

Portage Biotech and Biohaven Pharmaceutical announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to BHV-0223 for the treatment…

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US FDA grants orphan drug designation to BHV-0223 for spinocerebellar ataxia treatment – pharmabiz.com

Ataxia is a complaint of loss of control of voluntary body movements and can involve unsteady gait, speech difficulties, and clumsiness, potentially progressing…

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Portage’s Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia – PR Newswire (press release)

Vlad Coric, M.D., CEO of Biohaven, added, “Receiving the orphan drug designation request for spinocerebellar ataxia supports our global development strategy and goal…

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Promising Friedreich’s Ataxia Therapy Seen in Synthetic RNA and DNA Agents Used by UT Researchers – Friedreich’s Ataxia News

University of Texas (UT) Southwestern Medical Center researchers have identified RNA and DNA synthetic agents that increase frataxin levels and alleviate Friedreich’s ataxia…

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UT Southwestern scientists find potential treatment for Friedreich’s ataxia UT Southwestern Medical Center – EurekAlert (press release)

16, 2016 – Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich’s ataxia,…

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Retrotope Advances RT001 in Clinical Trials to Treat Friedreich’s ataxia – Marketwired (press release)

LOS ALTOS, CA–(Marketwired – Feb 22, 2016) – Retrotope, a privately held clinical stage pharmaceutical company, today announced the successful completion of the…

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Agilis Biotherapeutics and Waisman Biomanufacturing Enter Into Exclusive Manufacturing Agreement for Friedreich’s … – Business Wire (press release)

It is the most common inherited ataxia, with 1 in every 100 people being carriers of a mutated FXN gene. FA typically arises…

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Synthetic RNA and DNA could reverse protein deficiency that causes Friedreich’s ataxia – News-Medical.net

Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich’s ataxia, a neurological disease…

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Ride Ataxia’s Kyle Bryant to Be Keynote Speaker at Exposition – Friedreich’s Ataxia News

The Illinois Biotechnology Industry Organization (iBIO) recently announced that Kyle Bryant, founder and director of Ride Ataxia, Friedreich’s Ataxia Research Alliance, will be…

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In Friedreich’s Ataxia, Neurotrophins Hold Therapeutic Potential to Prevent, Treat Neurodegeneration – Friedreich’s Ataxia News

Researchers have demonstrated in the lab (in vitro) and in living subjects (in vivo), that gene transfer of brain-derived neurotrophic factor (BDNF) protected…

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Friedreich’s Ataxia Drug Developer RaNA Therapeutics to Present Data at Conference – Friedreich’s Ataxia News

RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in Friedreich’s Ataxia, announced…

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Spinocerebellar ataxia type 21 exists in the Chinese Han population – Nature.com

Recently, mutations in transmembrane protein 240 (TMEM240) were identified as the cause of spinocerebellar ataxia type 21 (SCA21) in several French families. Clinically,…

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Promising Friedrich’s Ataxia Drug Candidate Gains Support of NIH Scientists – Friedreich’s Ataxia News

Chondrial Therapeutics recently announced that further development of its lead drug candidate for the treatment of Friedreich’s ataxia, TAT-Frataxin (TAT-FXN), will be aided…

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Ely police officers to take on Three Peaks Challenge in aid of Ataxia UK – Ely News

All funds they raise will go towards the charity, Ataxia UK. Mark said: “We are all really excited about doing the challenge as…

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Friedreich Ataxia and Nephrotic Syndrome Case Studies Show Steroid Treatment Promising – Friedreich’s Ataxia News

FRDA is a genetic, progressive, neurodegenerative disorder that causes damage to the nervous system and leads to impaired muscle coordination (ataxia). The disease,…

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Avalanche to Expand Gene Therapy Holdings by Acquiring Annapurna – Friedreich’s Ataxia News

Avalanche Biotechnologies, based in Menlo Park, California, is a gene therapy company focused on the treatment of chronic or debilitating diseases, including Friedreich’s…

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Potential Friedreich’s Ataxia Therapy Advances with FARA’s Support – Friedreich’s Ataxia News

CAT-4001 is being developed by Catabasis as a potential treatment for neurodegenerative diseases, such as FA and amyotrophic lateral sclerosis (ALS). CAT-4001 is…

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