A study of an investigational drug called ZX008 in patients who have refractory Lennox-Gastaut syndrome has produced promising results, according to Zogenix. For more detailed information, you can view…
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 speciality pharmaceutical company Advicenne has announced that the US FDA has cleared the Investigational New Drug (IND) application submitted on behalf of ADV7103. ADV7103 is an investigational treatment for…
September is Primary Biliary Cholangitis Awareness Month! What is PBC? Primary biliary cholangitis (PBC) is a chronic disease of the liver characterized by a slow, progressive damage to the bile ducts…
I know that it's been months since Kylie Jenner tweeted that Snapchat was over, and the company stock dropped like a billion dollars over night, but if you, like me,…
According to a story from the Irish Examiner, treatment with the recently approved drug patisiran helped a British surgeon named Carlos Heras-Palou save both his hands and his career. Patisiran…
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…
McMaster University researchers investigating the factors underlying patient relapse in acute myeloid leukaemia believe that they may have found a type of cell that plays an important role. The researchers…
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 BioPortfolio, the drug development company Dicerna Pharmaceuticals recently announced the release of proof of concept data in its Phase I trial testing the company's experimental…
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…
September is Histio Awareness Month! That "histio" refers to histiocytic disorders, which are are a group of diseases that occur when there is an over-production of white blood cells known…
The companies BeiGene Ltd. and SpringWorks Therapeutics have announced that they will collaborate to research the safety and preliminary efficacy of a combination of two drugs: BeiGene’s investigational drug lifirafenib…
According to a story from Medpage Today, three recent research studies have managed to reveal new biomarkers for myelodysplastic syndromes. Biomarkers are a crucial factor for understanding a medical condition…
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 wect.com, when Beth Quinn received news of her diagnosis of ovarian cancer, she knew that it was only a matter of time before the disease…
Dova Pharmaceutics has submitted a supplemental New Drug Application to the US FDA on behalf of Doptelet® (avatrombopag), which is being researched as an investigational treatment of immune thrombocytopenia in…
The medical company Biogen has announced that the first patient has been enrolled in a Phase 3 clinical trial investigating a drug as a potential treatment for severe cerebral edema…
A recent study using mice suggests that Huntington’s disease may begin to show symptoms early on in life. For more detailed information, you can read the source article here, at…
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…
The US Department of Labor has shared an opinion letter that protects the job security of eligible living organ donors who need to take time off for their donation. For…
Content Warning: This article discusses sensitive topics of mental illness and suicide as it relates to rare and chronic illness. There's no easy way to talk about suicide. This article…
The US Food and Drug Administration and Eiger BioPharmaceuticals, Inc. have met to discuss lonafarnib, a drug being investigated as a potential treatment for Hutchinson-Gilford Progeria Syndrome. For more detailed…
Doctors in Britain are being urged to write outpatient clinic letters in plain English and address them to their patients, rather than using technical medical language written to the patient’s…
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