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…
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…
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…
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…
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…
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…
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…
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…
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"…
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…
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…
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…
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…
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…
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…
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…
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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,…
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…
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…
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…
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…
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…
According to a story from Market Screener, the biopharmaceutical company Biohaven Pharmaceutical Holding Company Ltd. recently announced that the first patient has been enrolled in the company's Phase 3 clinical…
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)…
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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