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Dystrophic epidermolysis bullosa (DEB) is a condition that has many sub-types, one of which is pretibial. The COL7A1 gene, which is responsible for producing VII collagen, is the cause of…
According to a story from 10tv.com, Kellan Shatto is an eight year old boy who really likes to play with Legos. He also has a rare disease: neurofibromatosis. The disease…
Amyloidosis is characterized by stiffness and an inability to function in certain organs. Because of the danger this presents, it is important that doctors are able to monitor the…
By Jack Gerard from In The Cloud Copy Medical response dogs are trained to recognize potential medical problems with their owners, alerting them or someone else when they detect a…
By Jodee Redmond from In The Cloud Copy University researchers Richard Terek and Qian Chen have published a paper drawing attention to a possible treatment for chondrosarcoma, which is a…
Autoimmune encephalitis is a group of diseases that was only identified about a decade ago. The newness of this condition makes it difficult to completely understand it. Dr Robb…
By Rachel Whetstone from In The Cloud Copy Jane Edwards is a 45-year-old woman from Stamford, Lincolnshire. She has a rare autoimmune disease called vasculitis. Her immune system attacks her…
Many artists draw inspiration from events in their lives. Dylan Mortimer is no exception, as his art is inspired by his cystic fibrosis. He creates art that mimics lungs,…
A PIONEER trial has been conducted in order to evaluate the effects of avapritinib, a drug meant to treat systematic mastocytosis. Researchers want to find a better treatment for…
According to a story from The People's Pharmacy, the US Food and Drug Administration (FDA) has given the green light for the use of generic drugs manufactured in India despite…
According to a story from ucb.com, the biopharmaceutical company UCB has recently released the final results from its phase 2 clinical trial which tested the company's investigational monoclonal antibody rozanolixizumab…
By Jack Gerard from In The Cloud Copy Travis Allen, of Lisbon, Iowa, was a painter who specialized in photorealistic works. Unfortunately, a rare autoimmune condition stole his eyesight…
According to a story from the Washington Post, rare diseases are a much more widely spread problem than you might think. There are at least 7,000 diseases that are considered…
The European Organization for Research and Treatment of Cancer (EORTC) has been evaluating zotiraciclib, which is a new treatment for glioma. This drug has recently been granted the Orphan…
According to a story from thejournal.ie, a recent study has discovered a new method for bringing down inflammation in cystic fibrosis. The study authors have concluded that the approach could…
Jojo, a ten-year-old girl, has become the first person to ever receive a new gene therapy for the treatment of GM1 gangliosidosis. The treatment, called AXO-AAV-GM1, was used in…
According to a story from Ankylosing Spondylitis News, the results of a recent phase 2/3 clinical trial indicate that the drug upadacitinib (marketed as Rinvoq) can be an effective treatment…
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Encouraging results from a clinical study were reported recently in Science & Enterprise describing a treatment for ALS using stem cells extracted from a patient’s bone marrow. The clinical…
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A recent article in Bloomberg’s SFGate focused on the price of drugs. The average patient cannot afford the price of many drugs now on the market. Insurance companies have…
Some scientists never have the opportunity to meet a single patient in the community of the disease they work their entire lives trying to understand. This can make them feel…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
By Jack Gerard from In The Cloud Copy. While vaccines are largely considered safe for most of the population, there are some people whose doctors recommend avoiding at least some…
By Jodee Redmond from In The Cloud Copy The results of a study have found that betaine and the active form of folic acid, L-methylfolate, are not effective measures to…
By Rachel Whetstone from In The Cloud Copy For millions of Americans who are affected by rare diseases, hope and health lies in the availability of plasma donations. One use…
The University of Massachusetts Medical School has recently announced preliminary results from their trial for Tay-Sachs disease. This trial is investigating a gene therapy and researchers are excited about it's…
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