I’m writing this article now because it was something that I needed one year ago and didn’t have. I didn’t know anyone who had moved abroad with Common Variable Immunodeficiency…
According to a story from BioSpace, the biopharmaceutical company Soleno Therapeutics recently announced that it will be updating the minimum age for participating patients in its Phase III clinical trial…
According to a story from techcentral.ie, the company OpenApp, which is based in Dublin, Ireland, has a developed a new online system that will allow for "virtual panels" of disease…
According to a story from The Guardian, the viral disease monkeypox has been reported in the UK for the first time. The first patient was diagnosed on September 8th and…
According to a story from Stat News, The US Food and Drug Administration (FDA) has recently sent a message to the pharmaceutical company Amicus Therapeutics in which they denies the…
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
Meet Ethan Ethan Faneran-Burley was diagnosed at two years old with Mucopolysaccharidosis type 6 (or MPS type 6). It's a progressive, degenerative, and ultimately fatal condition. Those with MPS type…
The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…
According to a story from stuff.co.nz., Janelle Brunton-Rennie and Kurt Brunton were celebrating their third wedding anniversary this past January. Little did they know that the next day, their lives…
According to a story from Vector, a patient in an early experimental trial for a potential treatment for PTEN hamartoma tumor syndrome appears to be benefiting from the therapy. Ten…
Misdiagnosis after Misdiagnosis Ever try to google your symptoms to figure out what’s wrong with you? I think we all have at some point. Is it a cold? A virus?…
The George Washington (GW) University School of Medicine and Health Sciences has announced that they and twelve other research centers are collaborating with the Scleroderma Research Foundation to launch the…
According to a story from the Harborough Mail, Magnus, who is six years old, was diagnosed with neuroblastoma around Christmas last year. Unfortunately, the disease had already spread and was…
A new clinical trial, which can be found here at clinicaltrials.gov, is slated to begin and is currently recruiting patients. In this trial, the investigational drug pacritinib will be tested…
According to a story from The Rheumatologist, a recent study of neurosarcoidosis is providing new information into how this disease develops, as well as how it can be categorized. This…
Who Lives With a Chronic Illness? A Lot of People. More than half of the world’s population lives with a chronic condition and of the 2 trillion the US spends…
According to a story from The Chicago Tribune, Darnesha Bankston, age 23, was rushed to the hospital just before Christmas last year with a bunch of severe, life threatening symptoms.…
According to a story from Shawnee Mission Post, the nonprofit organization RareKC is working to support people with rare diseases that are living the Kansas City area. When living with…
Happy Friday Everyone! As we get ready for the weekend, we want to spotlight four articles. We have an article about a man with Parkinson's who uses social media to…
According to a story from statnews.com, Dr. Julia Brody, who heads the Silent Spring Institute located in Newton, Massachusetts, says that her laboratory has always allowed patients and study participants…
According to a story from the Santa Cruz Sentinel, the earliest results from a critical gene editing study could offer some encouragement for those hoping to treat rare genetic diseases…
The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…
Another article previously published under "Featured Adult: Rebekah Palmer's Story" in The Cystinosis Advocate/ Volume 10, Issue 1/ Spring and Summer 2017: I feel an obstacle in being the adult patient…
According to a story from sectorpublishingintelligence.co.uk, a study from the Fraser Institute suggests that the Canadian government's initiative to more closely control the pricing of drugs could place access to…
The information in this article is sourced from this leaflet from Frambu and this publication from Eurordis. You can also visit Frambu’s website by clicking here. Frambu is a national centre…
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