Spinocerebellar Ataxia – Patient Worthy

FDA Rejects Rare Disease Treatment Despite Strong Clinical Data: Biohaven Challenges Regulatory Decision on Spinocerebellar Ataxia Drug

Post author:Bree Clare
Post published:November 20, 2025
Post category:Spinocerebellar Ataxia

Biohaven Pharmaceuticals faced a significant setback when the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) rejecting the company's New Drug Application for Vyglxia (troriluzole), a promising…

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Patient Story: Spinocerebellar Ataxia Has Affected My Whole Life

Post author:Patient Worthy Contributor
Post published:May 30, 2024
Post category:Spinocerebellar Ataxia

Written by Martha Harlam Until 1986 I led a wonderful life. I got to sing for my supper in musicals and opera. Now, spinocerebellar ataxia was taking control of my…

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ICYMI: After a 25 Year Search, a Doctor Discovers the Cause of Spinocerebellar Ataxia 4

Post author:Rose Duesterwald
Post published:May 22, 2024
Post category:Spinocerebellar Ataxia

You may not have heard of spinocerebellar ataxia 4 (SCA4). It is rare and it is a devastating movement disease. SCA4 generally begins when a person is 40 or 50,…

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Researchers Identify How GCC-Repeat Expansion in ZFHX3 Causes Spinocerebellar Ataxia Type 4

Post author:Jessica Lynn
Post published:May 13, 2024
Post category:Spinocerebellar Ataxia

In 2010, K. Pattie Figueroa of the Spencer Fox Eccles School of Medicine at University of Utah was approached by a family whose child had spinocerebellar ataxia type 4 (SCA4).…

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Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction

Post author:Jessica Lynn
Post published:February 19, 2024
Post category:Spinocerebellar Ataxia

What role do brain alterations play in the onset and progression of spinocerebellar ataxia type 6 (SCA6)? While previous studies highlighted cerebellar changes in individuals with SCA6, the underlying mechanisms…

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FDA Refuses to Review Biohaven’s Application for Spinocerebellar Ataxia Drug

Post author:James Moore
Post published:August 9, 2023
Post category:Spinocerebellar Ataxia

According to a story from Global Genes, the US Food and Drug Administration (FDA) has said that it will not take the pharmaceutical company Biohaven's recent New Drug Application under…

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Rare Classroom: Spinocerebellar Ataxia

Post author:James Moore
Post published:July 14, 2023
Post category:Spinocerebellar Ataxia

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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Potential Link Exists Between Gut Microbiota Imbalance and Machado-Joseph Disease (MJD)

Post author:Jessica Lynn
Post published:June 14, 2023
Post category:Machado-Joseph disease Spinocerebellar Ataxia

Past studies have shown that our gut microbiota, or the collection of microorganisms (bacteria, archaea, fungi, yeast, viruses) within our gut, plays an immense role in our health. Our…

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Bachelor Australia’s Megan Marx Shares Spinocerebellar Ataxia Diagnosis

Post author:Jessica Lynn
Post published:February 1, 2023
Post category:Spinocerebellar Ataxia

In the reality television world, Megan Marx is best known for her time on Bachelor Australia, Bachelor in Paradise, and The Challenge Australia. But Marx is about to be…

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AI Spurs Discovery of Novel Spinocerebellar Ataxia

Post author:Jessica Lynn
Post published:June 22, 2022
Post category:Spinocerebellar Ataxia

The future of healthcare is going to be changed and impacted by the use of artificial intelligence (AI), hopefully in beneficial ways. According to an article from McMaster University, researchers…

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Family Raises SCA3 Awareness

Post author:Jessica Lynn
Post published:January 21, 2022
Post category:Spinocerebellar Ataxia

The Klassen family is no stranger to the rare disease community. According to the Denver Post, Greg Klassen was first diagnosed with spinocerebellar ataxia type 3 (SCA3) nearly ten years…

geralt / Pixabay

New Optimism for Treating Machado-Joseph Disease

Post author:Jessica Lynn
Post published:October 11, 2021
Post category:Machado-Joseph disease Spinocerebellar Ataxia

Dimeric calpains are calcium-activated cysteine proteases which help to regulate cell function. If these calpains are overactive, it could lead to fibrosis (tissue scarring) and tissue damage. In the…

mohamed_hassan / Pixabay

VO659 for SCA Earns Orphan Drug Designation

Post author:Jessica Lynn
Post published:July 1, 2021
Post category:Spinocerebellar Ataxia

Within the United States, the FDA developed the Orphan Drug Act to incentivize companies to develop drugs and biologics intended to treat patients with rare conditions. In this case, "rare"…

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GeneDx is Expanding Genetic Testing for Forms of Hereditary Ataxia

Post author:Kendall Mason
Post published:February 8, 2021
Post category:Friedreich ataxia Spinocerebellar Ataxia

GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…

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

Post author:Jessica Lynn
Post published:February 5, 2021
Post category:Friedreich's Ataxia Progressive Supranuclear Palsy Spinocerebellar Ataxia

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…

New Test Developed to Better Assess the Efficacy of Spinocerebellar Ataxia Type 3 Therapies

Post author:Trudy Horsting
Post published:October 29, 2020
Post category:Multiple Sclerosis Parkinson's Disease Spinocerebellar Ataxia

Researchers at the Mayo Clinic have collaborated with the international community to develop a potential way to test for spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph Disease. Additionally, they…

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

Post author:Rose Duesterwald
Post published:August 24, 2020
Post category:Rare Disease Rett Syndrome Spinocerebellar Ataxia

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

Post author:Jessica Lynn
Post published:May 25, 2020
Post category:Amyotrophic Lateral Sclerosis Spinocerebellar Ataxia

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

Post author:Sunniva Bean
Post published:March 23, 2020
Post category:Rare Disease Rett Syndrome Spinocerebellar Ataxia

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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Study Links CHIP Mutations to Specific Symptoms in Spinocerebellar Ataxia

Post author:James Moore
Post published:November 12, 2019
Post category:Spinocerebellar Ataxia

According to a story from MedicalXpress, the results from a recent study are linking biochemical changes affecting a protein known as CHIP to a diverse array of clinical characteristics that…

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

Post author:Rose Duesterwald
Post published:September 11, 2019
Post category:Alzheimer disease type 3 Alzheimer's disease without Neurofibrillary tangles Spinocerebellar Ataxia

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

Post author:James Moore
Post published:June 13, 2019
Post category:Spinocerebellar Ataxia

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

Post author:James Moore
Post published:June 7, 2019
Post category:Spinocerebellar Ataxia

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…

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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation

Post author:James Moore
Post published:May 31, 2019
Post category:Spinocerebellar Ataxia

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…

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This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness

Post author:James Moore
Post published:May 21, 2019
Post category:Rare Disease Spinocerebellar Ataxia

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…

FDA Rejects Rare Disease Treatment Despite Strong Clinical Data: Biohaven Challenges Regulatory Decision on Spinocerebellar Ataxia Drug

Patient Story: Spinocerebellar Ataxia Has Affected My Whole Life

ICYMI: After a 25 Year Search, a Doctor Discovers the Cause of Spinocerebellar Ataxia 4

Researchers Identify How GCC-Repeat Expansion in ZFHX3 Causes Spinocerebellar Ataxia Type 4

Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction

FDA Refuses to Review Biohaven’s Application for Spinocerebellar Ataxia Drug

Rare Classroom: Spinocerebellar Ataxia

Potential Link Exists Between Gut Microbiota Imbalance and Machado-Joseph Disease (MJD)

Bachelor Australia’s Megan Marx Shares Spinocerebellar Ataxia Diagnosis

AI Spurs Discovery of Novel Spinocerebellar Ataxia

Family Raises SCA3 Awareness

New Optimism for Treating Machado-Joseph Disease

VO659 for SCA Earns Orphan Drug Designation

GeneDx is Expanding Genetic Testing for Forms of Hereditary Ataxia

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

New Test Developed to Better Assess the Efficacy of Spinocerebellar Ataxia Type 3 Therapies

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

Exploring the Similar Pathology “Genetic Doppelgängers” ALS and SCA36

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

Study Links CHIP Mutations to Specific Symptoms in Spinocerebellar Ataxia

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

Experimental Spinocerebellar Ataxia Treatment Earns Orphan Drug Designation

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

Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation

This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness

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