Ryan Csupak has been in an emergency room 30 to 40 times, unable to move his legs and arms. He’s experienced shortness of breath, extreme fatigue and weakness. This is the reality of having ...

Rare diseases, though seldom recognised and frequently misdiagnosed, affect approximately 36 million people in the EU.

ALP is an enzyme found in the blood that helps break down proteins. It plays a role in numerous processes in the human body, and any abnormalities in blood concentrations—either high or low—may ...

Recent advances in AI, including machine learning, natural language processing, and deep learning models, are transforming ...

Clinical trials are notoriously expensive, with operational expenses ranging from $50 million to over $115 million. 1 The ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...

X-linked hypophosphataemia (XLH) is a rare, hereditary and lifelong renal phosphate wasting disorder caused by mutations in ...

Cell and gene therapies (CGTs) represent the cutting edge of drug discovery. 1 These are amongst the most advanced medical treatments, specifically designed to correct the root genetic cause of an ...

25 February 2024 | Orphanet Journal of Rare Diseases, Vol. 19, No. 1 Homeopathic Pyrogenium Ointment as Adjuvant in Treatment of Traumatic and Infected Myiasis by Cochliomyia Hominivorax (Diptera ...