She Survived the Lebanese Civil War and 30 Years of Challenges to Help Rett Syndrome Patients

Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…

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Exploring the Similar Pathology “Genetic Doppelgängers” ALS and SCA36
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Exploring the Similar Pathology “Genetic Doppelgängers” ALS and SCA36

Recently, scientists from Emory University set out to discover whether there was any pathological link between spinocerebellar ataxia type 36 (SCA36) and amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). Both…

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After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome
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After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome

As originally reported in Discover Magazine, Huda Zoghbi took an unexpected route to winning the Breakthrough Prize in Life Sciences in 2017 for her discoveries of the genes responsible for…

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Alzheimer’s Disease: A Long Journey and Another Step Forward

  According to a recent article in MedicalXpress, the newest discovery in the search to reduce the risk of AD was through the work of researchers led by Dr. Jaehong Suh,…

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Experimental Spinocerebellar Ataxia Treatment Earns Orphan Drug Designation
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Experimental Spinocerebellar Ataxia Treatment Earns Orphan Drug Designation

According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…

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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation from the FDA

According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and…

Continue Reading Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation from the FDA
Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation
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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation

According to a story from BioSpace, the drug developer Cadent Therapeutics recently announced that its investigational product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…

Continue Reading Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation
This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness
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This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness

According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…

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Exciting Gene Therapy Progress in Huntington’s Disease and Spinocerebellar Ataxia

Huntington's Disease & SCAs  Huntington's disease (HD) and spinocerebellar ataxia (SCA) are both neurodegenerative diseases caused by the same type of error in the body's DNA. Essentially, the CAG or…

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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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30 Million Euros Allocated to Fund Precision Medicine Research Projects for Neurodegenerative Diseases

Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…

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New Orphan Drug Designation for Potential Spinocerebellar Ataxia Treatment
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New Orphan Drug Designation for Potential Spinocerebellar Ataxia Treatment

Spinocerebellar Ataxia (SCA) refers to a group of neurodegenerative disorders. Ataxias are a component of the nervous system which control movement. SCA occurs when the ataxias in the brain or spinal…

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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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Clinical Trials Begin for a Possible Spinocerebellar Ataxia Treatment
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Clinical Trials Begin for a Possible Spinocerebellar Ataxia Treatment

According to a story from pharmavoice.com, the pharmaceutical company Cadent Therapeutics has announced the beginning of the Phase I clinical study for its drug candidate CAD-1883. The therapy is a…

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First-in-Class Drug May be Coming to You, SCA Patients!
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First-in-Class Drug May be Coming to You, SCA Patients!

Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA)…

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