According to a publication from Medicine Matters, a 12-person task force organized by the European League Against Rheumatism (EULAR) has released a set of 12 recommendations to improve quality of…
According to a story from news-medical.net, an international research team affiliated with the UK Medical Research Council, Indiana University School of Medicine, and the University of Kansas have released a…
According to a publication from Fierce Biotech, Biotech company Sangamo recently updated investors on three trials it has underway testing experimental forms of gene and cell therapies. The trials are…
According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…
According to a story from Science Daily, a recent study has found that lithium chloride was able to improve muscle size and strength in a mouse mode of a specific…
Functional neurological disorder is a conversion disorder. This type of complex disorder is often thought to be linked with psychological symptoms, serious stress, or mental distress. With functional neurological…
According to a story from bioengineer.org, a team of researchers associated with Oregon State University are making further headway in understanding the connection between our health and the bacteria that…
Stem Cell Therapies While stem cell therapies hold a great deal of promise for many rare diseases, there is still a lot that we don't understand. Experimental stem cell therapies…
Do you have ADPKD? If you or someone you know has ADPKD, email us at [email protected]. We'd love to hear from you about your experience! According to a story from…
According to a story from ANCA Vasculitis News, a recent study conducted in Korea determined that ANCA vasculitis patients in the region were at a greater risk of developing thyroid…
"A new way to improve the efficacy of a drug taken by millions of patients throughout the world." Background Adalimumab (also known as Humira) and infliximab (also known as Remicade)…
MVID Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID…
According to a story from Science Nordic, the monoclonal antibody drug rituximab (often marketed at Rituxan) had no effect on patients with chronic fatigue syndrome (CFS). Chronic fatigue syndrome is…
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc. has recently announced that it has opened enrollment for its phase 1/2 clinical trial. This clinical trial…
According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
According to a story from mdmag.com, a recent study that was presented at the 2019 meeting of the American Society of Clinical Oncology has demonstrated the potential of a two…
A recent study published in PLOS Biology has shown a promising new vulnerability within human viruses which could lead to the development of a new antiviral drug. This study was…
According to a story from Scleroderma News, a team of researchers associated with Michigan Medicine has been awarded a grant to the tune of $10.2 million from the National Institutes…
Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…
According to a story from Market Screener, the drug developer Reviva Pharmaceuticals, Inc. recently announced that it has completed a recent meeting with officials from the US Food and Drug…
Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…
According to a story from home.cableone.net, the biopharmaceutical company Takeda Pharmaceutical Company Ltd. recently announced the latest updates to their phase 3 clinical trial which is testing the company's drug…
Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…
According to a story from statnews.com, a recent study found that the Affordable Care Act was able to help reduce differences in access to critical cancer treatment between black and…
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