Vicki and Fred Modell have spent years since their son’s death in the 90's advocating for more research and assistance to be provided to those with primary immunodeficiency disease. Their…
.jpg)
Parkinson’s disease can be a hard disease to manage. Not only does it take a physical and emotional toll on patients and loved ones, but managing every day activities can…
.png)
Major European and international kidney associations have joined in an awareness campaign to stress the urgent need to include patients with chronic kidney disease in clinical trials. In a recent…
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shroud…
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…
Phenylketonuria (PKU) is a rare disease which causes phenylalanine (an amino acid) to build up in the body, leading to serious health complications. Since amino acids are obtained through the…
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
The Study Juvenile idiopathic arthritis (JIA) is a rare disease that causes inflammation in the joints. It is of unknown origin and has no cure. Previous investigations in studies with…
According to a press release from the New Jersey-based biotechnology company Cellectar Biosciences, the Food and Drug Administration (FDA) has granted "Fast Track" designation to the Company's experimental diffuse large…
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…
.jpg)
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
