According to a story from Benzinga, the drug company Abeona Therapeutics, Inc. has recently announced plans to move forward with its phase 3 clinical trial following the lifting of the…
Grajevis Bakatunkanda lived in the Democratic Republic of the Congo, where he received treatment for malaria after experiencing weekly pain crisis. The treatment he received did not work, as…
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…
A new study has found that young people who have inflammatory bowel disease (IBD) are five times more likely to develop an infection than the general population. They also…
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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 Caitlin Seida from In The Cloud Copy Although it’s already known that the cause of Alexander disease (AxD), a type of leukodystrophy, is a misfolded protein known as GFAP.…
According to a story from statnews.com, the family of 46 year old Tammy Stewart has been plagued by Huntington's disease, an ultimately fatal, progressive, degenerative disease that causes decades of…
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Since most people cannot truly understand the challenges faced by a blind person, then an artist who cannot see is unimaginable. According to a recent article in the British…
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…
By Jack Gerard from In The Cloud Copy When it comes to the diagnosis and treatment of diabetes, many consider this to fall under the role of a primary care…
According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…
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…
According to a story from globalnews.ca, Andre and Joshua Laroque are nine and seven years old respectively. The brothers, being close in age, spend a lot of time together and…
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…
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,…
Researchers from Brigham and Women's Hospital recently announced a new discovery which could potentially lead to a new multiple sclerosis (MS) therapy. This research has been published in Cell Host & microRNA.…
Therapix Biosciences specializes in cannabinoid pharmaceuticals. Currently, they are working on developing a therapy called tetrahydrocannabinol or THX-110 as a treatment for Tourette syndrome (TS). Additionally, it is being developed…
Former Bachelorette star J.P. Rosenbaum announced on Instagram that he has been diagnosed with rare Guillain-Barré syndrome (GBS). Whether you're a fan of the Bachelor/Bachelorette franchise or this is the…
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…
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…
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