According to a story from thenewsreports.com, the biopharmaceutical company XORTX Therapeutics, Inc., recently announced that the company and its partner Cato Clinical Research have received correspondence from the US Food…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…
Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.…
Tay-Sachs disease and Sandhoff disease (also known as GM2 Gangliosidosis), and GM1 Gangliosidosis are neurodegenerative conditions. They are a result of a defective enzyme. This enzyme causes toxic gangliosides to accumulate within the…
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…
The Myasthenia Gravis Foundation of America (MGFA) recently conducted a survey which found that most myasthenia gravis patients are not satisfied with their current treatment regime. These patients wanted a therapeutic…
Familial adenomatous polyposis (FAP) is a condition which causes extra tissue to form in various parts of the body such as the large intestine and gastrointestinal track. FAP affects about…
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