There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
The holiday season and end of the year/decade is upon us! As schedules slow down and we begin to reflect, I too began to take stock of the experiences that…
According to a story from Medical Xpress, a recent study has confirmed the status of palmoplantar pustolosis as a rare, orphan disease. A recent study of patients with the disease…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
By Caitlin Seida from In The Cloud Copy 2019 was a record year for drug recalls due to safety and quality concerns. It’s also been a record year for the…
By Caitlin Seida from In The Cloud Copy Medical alert dogs can give their companions a newfound sense of independence and freedom. Enter Bill, a golden retriever owned by Laura…
Treatment options for Gorlin syndrome are often surgical, meaning that patients face possible scarring, loss of facial function and structure, and lowered quality of life. PellePharm aims to change…
By Rachel Whetstone from In the Cloud Copy Pete Frates, known for being an Amyotrophic Lateral Sclerosis (ALS) advocate and an inspiration for the Ice Bucket Challenge, has passed away…
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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…
Philippe Pakter’s daughter Lysiane was born on March 29th 2017 in Lyon, France. However, soon after her birth, Philippe and his partner Delphine were hit with shocking news: Lysiane was…
Happy Thursday, Patient Worthy Friends! This week we're highlighting two stories on families who went on a diagnostic odyssey to understand their children's rare diseases. Additionally, we have some big…
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…
By Rachel Whetstone from In The Cloud Copy. Rheumatoid Arthritis (RA) causes pain and suffering to patients around the globe, and it can be difficult to diagnose in the early stages.…
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…
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…
As originally reported in Angelman Syndrome News, Terry Jo Bichell is a PhD researcher, former director and scientific officer of the Angelman Biomarkers and Outcomes Measures Alliance and Tennessee's ambassador…
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…
Pulmonary Hypertension Association The Pulmonary Hypertension Association (PHA) is headquartered in Silver Spring, Maryland. It was formed in 1991 by 4 women passionate about finding a cure. Additionally, they hope…
Copyscape score: 6% Currently there are only two FDA approved treatments for the treatment of amyotrophic lateral sclerosis (ALS). These treatments are only modestly effective, partly because ALS is not…
As originally reported in Medical Xpress, CRISPR has been a hot conversation starter in recent years. It has been framed as gene editing made easy: cheaper and simpler than previous…
According to a story from BioPortfolio, Acceleron Pharma and the Bristol-Myers Squibb Company have announced recently that the US Food and Drug Administration (FDA) will review the supplemental Biologics License…
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