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…
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…
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 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,…
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.…
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…
Alex Barker never planned on marrying someone with his same rare diagnosis, but he has to admit, the shared experience has made him and his wife closer. Alex has Moebius…
The most common treatment for autoimmune conditions such as ankylosing spondylitis, plaque psoriasis, Crohn's disease, juvenile idiopathic arthritis, and rheumatoid arthritis is currently a drug called Humira, created by AbbVie.…
"Something's just not right - this is beyond coincidence." Ocular Melanoma is a rare cancer which only affects 5 out of every one million people. Typically, it affects older white…
The longer patients live without a diagnosis, the longer they are living without the proper care and the proper treatment. The problem is twofold when a patient is diagnosed incorrectly.…
The Patient-Centered Outcomes Research (PCORI) The PCORI is a nonprofit whose primary main mission is to help support research that will allow patients to make informed healthcare decisions. What makes…
Cassie Bebout is an Auburn University undergraduate student, on track to graduate this May. She chose the school for one primary reason- it is the home of Dr. Doug Martin…
Happy Wednesday Everyone! We hope everyone had a good St Patrick's Day. Today, we're highlighting a story from PW contributor Denise Crompton on grief. This is followed by an article…
The book Five Feet Apart was first released to the public in November 2018 and on March 15th, 2019, a film based on the novel was released into theaters. The…
Shifting Contracts Over the years the United States healthcare system has shifted more toward reimbursement models that are outcome-based instead of service-based. In essence, this means that instead of paying…
Rett Syndrome Rett syndrome is a rare and progressive neurodevelopmental disorder caused by mutations to the MECP2 gene. While it affects all ethnic and racial groups, it is found almost…
Progeria and Atherosclerosis Progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS), is a rare disease affecting just 400 people across the world. It is caused by a mutated LMNA gene.…
Phase 1 Trial A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin's lymphoma who have not responded…
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from dubainewstoday.blogspot.com, the data from a Phase I clinical trial suggests that an experimental drug candidate from the pharmaceutical company Boehringer Ingelheim could substantially improve the…
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