August 3rd is CLOVES Syndrome Awareness Day! The CLOVES Syndrome Community (CSC) is an organization that "supports, educates, empowers and improves the lives of those affected by CLOVES Syndrome" and…
The struggle of living with a rare disease and dealing with insurance issues was in the spotlight on national television. During Tuesday night's 2020 Democratic debate for the president, Senator…
A Colorado mom is on a mission to raise money for her daughter's type 1 spinal muscular atrophy (SMA) treatment -- that would save her child’s life. Let's hear little Maisie…
In a time where we get bombarded by all sorts of troubling news -- it's so soul-refreshing to see these truly inspirational moments go viral. A video of limb different,…
In an exclusive "Good Morning America" interview this week, Dr. Priscilla Chan announced the launch of Rare As One, a new program from the Chan Zuckerberg Initiative where she is co-CEO…
Last week, the New England Journal of Medicine published a report about a rare, disease-like infection that is a real cautionary tale. A 41-year-old British woman was diagnosed with acanthamoeba keratitis…
Some promising news from across the pond! A new international consortium funded by the European Union will focus on speeding up the diagnosis and development of rare diseases and treatments.…
Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF). This…
15-year-old George Taylor reached out to NY Giants Running Back Saquon Barkley, telling the star NFL player how he's an inspiration to him for when he undergoes his chemotherapy for Ewing's sarcoma,…
Cleveland Browns wide receiver Jarvis Landry has been affected personally by CF; he lost his high-school sweetheart Mya Zimmer in July of 2015 after her long battle with the rare…
Meet the drag queens and king of Drag Syndrome -- a group of Brits with Down syndrome who love performing in drag. And we're here for it! Some might not…
Meet Reece Anthony Rubino - a 13-year-old teen who loves video games, Disney World, and baseball. At just 3 months old, he was diagnosed with Type 1 spinal muscular atrophy…
Meet 6-year-old Grayson Kole Smith -- who after 36 surgeries and a grim prognosis has overcome the odds to live his life. Grayson was born with several serious medical issues…
A local Utah newspaper spotlighted 40-something nurse Tammy Henry, whose fibromuscular dysplasia not only can't stop her from running in her city's half marathon; but inspired her to be healthier. Behold,…
July is Gastroschisis Awareness Month! Thanks to the great work of the Avery's Angels Gastroschisis Foundation, there are a number of ways and resources to make the most out of…
The San Francisco Chronicle had a pretty neat story about how one man's post-cancer neurological issues lead to a so-rare-it's-still-unnamed disease diagnosis. And tracking how we got to the movement…
Everyone processes their rare disease (or really ANY disease) diagnosis differently. When 23-year-old Blaide Woodburn was diagnosed with phenylketonuria, it sparked in him an interest in health and science that…
Any Noles fans? Well, prepare to love them even more. Last weekend, players on the Florida Statue University Seminoles football team participated in FSU’s annual “Lift for Life” event, which raises…
July is national Cleft And Craniofacial awareness and prevention Month! While one of the fruits of summers is time off from school, shortened summer hours, or sunny vacations - it's…
Some of us here may have heard of Camp Sunshine -- which provides retreats "combining respite, recreation and support, while enabling hope and promoting joy, for children with life-threatening illnesses…
This is a great story about people coming together for a young boy with a rare disease, reported by a local tv news affiliate. Members of the Rhode Island State…
Did anyone catch this AMAZING kid on 'America's Got Talent' a few weeks ago? Tyler Butler-Figueroa -- an 11-year-old from North Carolina -- absolutely slayed his violin performance during the…
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…
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…
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