Cambridge, Mass. – Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades. But decades can feel impossibly far off to someone who currently has a fatal disease....
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NOVATO, California — The Phase 2b clinical trial will enroll up to six cystinosis patientswith a history of good compliance using the currently availableimmediate-release form of cysteamine bitartrate, the only drug cleared formarketing by the U.S. Food and Drug Administration (“FDA”) and EuropeanMedicines Agency (“EMEA”) to treat cystinosis. The clinical...
Ottawa, Canada – How can someone have alcohol intoxication without consuming alcohol? Auto-brewery syndrome, a rare condition in which gut fungi create alcohol through fermentation, is described in a case study in CMAJ (Canadian Medical Association Journal) https://www.cmaj.ca/lookup/doi/10.1503/cmaj.231319. “Auto-brewery syndrome carries substantial social, legal, and medical consequences for patients and...
DURHAM, N.C. — The Rare Cancer Research Foundation (RCRF) is proud to announce an exciting collaboration with Spanios aimed at propelling the discovery and validation of groundbreaking therapeutic options for rare cancers. This partnership, facilitated through RCRF’s innovative initiative, Pattern.org, marks a significant step towards addressing the pressing need for...
Basel, Switzerland – The rare case of a man with Lambert-Eaton myasthenia syndrome (LEMS) that co-occurred with non-Hodgkin lymphoma, a type of immune cell cancer, was described in a recent report. “In this case report, we present the case of a 57-year-old male diagnosed with non-Hodgkin’s lymphoma (NHL) who developed...
CHENNAI — When Latha could not wash utensils or even pick up a handkerchief from the floor, she did not realise that she was suffering from takaysus arteritis, ‘pulseless disease’ a rare disorder that occurs in Asian women. Surgeons at the city-based M R Hospitals — Dr S Saravanan, Dr...
It was only dumb luck that Jason Morgan found out about his illness. He experienced frequent muscle pain but without any remedy. Years of tests, blood draws, and unanswered questions only yielded an answer after Morgan’s doctor connected the dots between his mother and his aunt. He had Burton Muscular...
There are more than 7,000 rare diseases identified in the world today. While “rare” means that less than 200,000 people in the US are affected, to suffer from a rare disease is not actually rare at all. Around 10% of the American population suffers from a rare disease. A shocking...
When we think about common diseases or health conditions, diabetes, heart diseases and the likes top the list. Rare Disease Day, which falls on the last day of February, is all about raising awareness for rare diseases. This year, February 28 will see people raising awareness about diseases that aren’t...
Globally coordinated by The European Organization for Rare Diseases (EURORDIS), and over 65 national alliance patient organisation partners, Rare Disease Day aims to increase awareness and promote necessary changes for the 300 million people worldwide living with a rare disease. Over 600 events will be taking place this year in...