According to a story from Buzzfeed News, Sharon Greenstein, who lives with the rare autoimmune disease known as Sjögren's syndrome, can no longer find the drug that she uses for…
According to a story from The Intercept, Gilead Sciences' antiviral drug remdesivir, which is being tested as a treatment for coronavirus/COVID-19, was just given Orphan Drug designation by the US…
As originally reported in European Pharmaceutical Manufacturer Magazine, Chiesi, an Italian pharmaceutical company, has announced they will be launching a new division for rare disease research and therapy innovation. The…
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…
By Jodee Redmond from In The Cloud Copy Frankie Hewitson spent more than her fair share of time in the hospital during her childhood. The 27-year-old was diagnosed with three…
The American Kidney Fund (AKF) has recently announced the formation of a Coronavirus Emergency Fund intended to support low income dialysis and transplant patients who may face unanticipated financial challenges…
It's easy to feel helpless right now. You're told to stay home and just wait it out. So you're stuck feeling isolated and angsty, knowing there's nothing you can personally…
Chronic granulomatous disease (CGD) is a rare form of immunodeficiency. The most common form of this disease is X-linked chronic granulomatous disease (X-CGD) which is caused by a mutated CYBB gene. This…
The Fighting HARD Foundation Rare diseases are difficult to face for a myriad of reasons. They can be financially burdensome, physically painful, and emotionally draining. Further, they can make patients…
We know that older individuals and those who are immune compromised are more at risk for experiencing severe Covid-19. These include multiple sclerosis (MS) patients. Multiple Sclerosis is a rare…
According to a story from The Scarborough News, brother Connor and sister Kiera Pickering, aged eleven and twelve years respectively, will be some of the first patients to be treated…
Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…
A new Phase 1 clinical trial for celiac disease has just begun dosing patients. This trial is being run by Anokion SA. Celiac Disease Celiac disease is an autoimmune condition…
Pyoderma gangraenosum (PG) is a rare auto inflammatory disease which affects the skin. It unfortunately has no currently approved therapies. However, recent preliminary results from a Phase 2a trial have…
According to a story from NeurologyLive, patients being treated for autoimmune diseases such as multiple sclerosis (MS) or myasthenia gravis could be at heightened risk of infection with coronavirus because…
According to a story from BioSpace, the testing of drugs in clinical trials to address the current coronavirus/COVID-19 pandemic will soon be under way. The biopharmaceutical company Sanofi and the…
According to a story from Independent, a team of scientists from Canada have isolated the strain of coronavirus that has rapidly spread around the globe in recent months. What this…
According to a story from Healio, researchers are hard at work determining who could be at the greatest risk from infection with COVID-19/coronavirus. So far, the elderly, people with heart…
As originally reported in Biospace, there’s good news today for adult patients with acute hepatic porphyria in Europe, a genetic disorder which causes pain and paralysis. The European Commission has…
According to a story from businesswire.com, the Bristol Myers Squibb Company recently released the topline results from its phase 3 clinical trial. This study was testing a combination treatment consisting…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
According to a story from Financial Buzz, the biopharmaceutical company Mereo BioPharma Group plc recently announced that it has successfully concluded its Type B End-of-Phase 2 meeting with the US…
An issue with current treatments for Parkinson's disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
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