Zogenix has just announced that they have not only finished enrollment in their Phase 3 investigation of Fintepla for Lennox-Gastaut Syndrome (LGS), but that the final patient has been randomized…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. The event will take place in Seattle, Washington on August 10th, 2019. When: Saturday, August 10,…
The 2019 NORD Living Stronger Forum was held in Houston, TX, at the end of June. The event brought families and physicians from all over for educational presentations, community, and…
According to a press release from the Massachusetts-based biotechnology company Avrobio, Inc., clinical tests of the Company's experimental Fabry disease gene therapy, AVR-RD-01, have yielded impressive preliminary results. Though the…
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,…
Terri considers herself “very lucky”. In the 80s her paternal grandmother and her grandmother’s two sisters were diagnosed with ovarian cancer. In the 90s, her family entered into the BRCA1…
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…
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The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports that…
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…
Antiphospholipid syndrome (APS) causes various complications, one of which is thrombosis. Researchers are looking at ways to assess an APS patient’s risk of thrombosis, which covers various factors. Treatment will…
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…
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 Real World Health Care, nonalcoholic fatty liver disease and its less common, more serious form called nonalcoholic steatohepatitis (NASH), are silent but deadly conditions that…
According to a story from Pharmafield, The UK's National Institute for Health and Care Excellence (NICE) has recently approved a first in class gene silencing therapy for coverage on the…
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