When one rare disease is found to be in some way similar to another rare disease, research can often be accelerated for both. For instance, recent discovery by a professor…
According to a story from fortune.com, the French drug startup Pharnext, led by CEO Daniel Cohen, recently made an announcement not to long ago that made its stock shares rocket…
Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…
Extremely positive results have just been announced from a 5-year follow-up study of a systemic juvenile idiopathic arthritis (sJIA) treatment called Kineret. This treatment has been approved for use in…
M ost of the time, Whitney Weldon doesn’t think about her second skeleton. She was 9 the first time it really flared up. Bridges of bone grew from nowhere to…
According to a story from Gaucher Disease News, life for 24 year old Madeline Collin, who has Gaucher disease, has never been easy. However, being subject to bullying and a…
According to a story from globenewswire.com, the biopharmaceutical company Strongbridge Biopharma plc, recently presnted a new analysis of data from the company's Phase 3 trial. This trial was testing the…
Farber Disease Farber Disease is a rare condition otherwise known as Acid Ceramidase Deficiency. It is a lysosomal storage disease caused by a missing ceramidase enzyme. This is due to…
418 words 9% vs 744 words 5%Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune DiseasesAccording to a recent article in the EurekAlert researchers used data from…
According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…
According to a story from finanzen.ch, the drug companies Pfizer and Merck recently released a joint statement in which they announced that the companies' Phase III clinical trial would be…
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A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…
According to a story from the Clarion Ledger, Keith Bradshaw of Bolton, Mississippi was on his way to celebrate New Years Eve in 2015 when he was suddenly struck by…
A young girl in Mauritius, an island in the Indian Ocean, has recently been given a second chance at a normal life after receiving a rare disease diagnosis and…
According to the publication over the weekend from San Francisco CBS affiliate KPIX, a groundbreaking clinical trial for those with aromatic l-amino acid decadboxylase (AADC) deficiency was undertaken at UCSF…
According to a publication from EurekAlert, high-cholesterol patients, who do not take statins (a type of cholesterol-lowering medication often prescribed by doctors) due to adverse side effects may benefit from…
Though it may surprise you, according to an article from CURE magazine, there’s a lot that makes solid tumors in children different from solid tumors in adults. They can be…
According to a publication from Scleroderma News, the first patient in the study of experimental cutaneous systemic sclerosis (SSc) drug AVID200 has been enrolled in the ongoing phase 1b clinical…
According to a story from the Inter Press Service, the First Latin American and Caribbean Assembly of Organisations of People Affected by Hansen's Disease has just recently concluded. Hansen's disease,…
According to a story from ctvnews.ca, a recent treatment with gene therapy appears to have changed the life of thirty year old Josh McQuillin, who has a urea cycle disorder,…
Alpha-1 Antitrypsin Deficiency Alpha-1 Antitrypsin Deficiency (AATD) is a rare disease caused by low levels of the Alpha-1 Antitrysin protein. It can lead to lung disease and liver disease. Liver…
According to a story from The Rheumatologist, there still remains much to be learned about the mysterious rare disease antiphospholipid antibody syndrome, which is also known as simply antiphospholipid syndrome.…
According to a story from Neuroscience News, a team of researchers based out of the University of Copenhagen have made an intriguing discovery that could give new insight into the…
Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…
Cystic Fibrosis Cystic Fibrosis (CF) is a rare disease caused by a defected CFTR gene. It results in a buildup of mucus which impairs breathing and makes the lungs susceptible…
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