The NIH has awarded Geisinger Health System, along with the University of Washington in Seattle and Washington University in St. Louis, a $5.8 million, five-year grant to study the role of…
Rare disease drug development, which now accounts for nearly one-third of all drugs in active research & development worldwide, presents challenges that will accelerate the adoption of new development strategies…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
Happy 7-11 Day! We hope everyone's on their way to get their free slurpie today! For this week's editor's choice, we're highlighting four articles. First, we discuss kratom, the controversial…
According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…
According to a story from Ankylosing Spondylitis News, a recent study has found that the class of drugs known as tumor necrosis factor (TNF) inhibitors may actually be safer for…
It was announced this Monday that Fulcrum Therapeutics, a Phase 1 biotech developing therapies based on gene regulation for rare diseases, announced terms for its initial public offering (IPO), aka its…
According to a story from Wellcome Center Mitochondrial Research, a multi-center collaborative study that involved researchers from different sites around the UK has resulted in an improved understanding of the…
According to a publication at Markets Insider, the Food and Drug Administration (FDA) recently approved Soliris (eculizumab) as a treatment for neuromyelitis optica spectrum disorder. The approval marks the first…
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Gov.UK recently published a speech by Baroness Blackwood in which she announced that the National Health Service (NHS) is currently involved in one of the most significant transformations in social…
Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…
According to a story from PR Newswire, the drug selinexor (marketed as Xpovio), which was developed by Karyopharm Therapeutics, was recently approved by the US Food and Drug Administration (FDA)…
Growing up "Normal" Growing up, Steph Derham always thought she was normal. She says she has her parents to thank for that. Steph is incredibly grateful that her mom and…
Rare Disease Fund Many families affected by rare diseases cannot afford the necessary treatments. Due to the small population of people these diseases affect, exorbitant price tags are often placed…
This bit of 2017 news flew under the radar! But it's worth shining a spotlight! In 2017, Macedonia became the first country in the WORLD to issue a postage stamp…
According to a story from Market Screener, the biopharmaceutical company Theranexus and the Beyond Batten Disease Foundation (BBDF) have recently signed an agreement to work towards the continued development of…
According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the…
The FDA has approved a new therapy for chronic immune thrombocytopenia (ITP). This treatment is an oral therapy to be taken with food, created by Dova Pharmaceuticals. It is called…
Frankie Jr. Muretic Frankie Jr. Muretic lives in Mississauga in Ontario, Canada. One morning he woke up with a rash, causing his parents concern. They brought the five-year-old boy to…
According to a story from news-medical.net, a team of scientists affiliated with the Swedish Karolinska Institutet have developed a new method for categorizing patients with the rare inflammatory bowel disease…
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The FDA is putting patients with rare diseases front and center knowing that these patients have little support and in many cases cannot find specialists to diagnose and treat their…
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