Although Anabelle, a girl from the Phillipnes, is only seven, she has seen some of darker corners of the world. Just two years ago, she was held captive by a…
Sometimes, the only thing a rare disease needs is for technology to catch up with the times and save the day. Recently, this came in the form of three dimensional…
Finding a community is important for everyone -- but particularly if you have a chronic or rare disease. For the approximately 300,000 Americans with scleroderma, there's an active support group…
During a charity event, a proud UK father pushed his daughter on her wheelchair for 21 miles to raise money for her rare disease. Connie Elson is only 9-years-old, but…
Nerves were high last week as the Senate geared up to vote on the bill to overhaul the U.S. tax code. Members of the rare disease community felt particularly vulnerable,…
Leon "Lee" Garfat is a survivor. The young boy was just 4 months old when doctors told his parents he wouldn't live past 2. He had been diagnosed with a…
I want to be Isaiah Acosta when I grow up. The 17 year-old from Phoenix, Arizona was born without a jaw, due to the very rare disease situs inversus, which…
Happy Friday Patient Worthians! Gotta love that hectic-catch-up-crazy feeling post-Thanksgiving and pre-December holidays! We have heartwarming story of a little girl getting the ultimate gift for the holidays. We also…
Some people with Marfan syndrome look the part. They may be unusually flexible, with long arms, legs, and fingers, and a tall, willowy build. Some patients have scoliosis, crowded teeth,…
Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…
Glenda Thompson is a proud grandmother who will stop at nothing to ensure her grandson lives a comfortable life. 4-year-old Cole Thompson is suffering from a rare genetic disorder called…
4-year-old Colt Cosper is coping with a life-threatening disease with his new buddy, Spider Man. Shortly after his third birthday, Colt was hit with the hard diagnosis: myoclonic-astatic epilepsy. This…
Jocelyn Duff didn't expect to feel the contractions as early as she did. She wasn't due for another two weeks. But this is something they were ready for. With the…
Shani Dhanda, 31, was at a loss-- she had sent hundreds and hundreds of job applications, and no one even interviewed her. The Birmingham woman stands at 3'10". She was…
The Leukemia Research medical journal published results from a study that showed efficacy when treating Acute Myeloid Leukemia (AML). Great news for the rare disease community! Onconova Therapeutics announced the publication of results…
While President Trump hopes to overhaul tax credits, Arkansas patient advocate Andrea Taylor is trying to rescue a tax credit that benefits the rare disease community. Taylor's 9-year-old son, Aiden,…
Hofstra is one college campus that gives students with disabilities the support and accommodations they need. They even have their own program called Student Access Services (SAS). The program is…
The HLN channel aired a documentary special regarding the rare and mysterious condition, fatal familial insomnia (FFI). FFI is a very rare genetic sleep disorder, with less than 40 families diagnosed…
Good news for patients with Mucopolysaccharidosis Type VII (MPS VII)! In mid-November, the FDA approved a new drug for treatment of this rare disorder. The drug can be used for…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, affects approximately 30,000 people in the United States. The National Organization for Rare Diseases estimates that 5,000 more people are diagnosed…
Julian Randle always wanted to play basketball. Now, the 11-year-old suffering with a rare disease will be joining a college team in Kansas. It was almost like a dream. The…
Love them, hate them, or love to hate them - celebrities are a big part of our culture. They sell us clothes, perfumes, lifestyles - and some we even elect…
As the United States Senate gears up to vote on whether to pass their version of the Republican-crafted tax reform legislation (the House of Representatives has already passed their version),…
If you watch "Eastenders" on the BBC, then you're familiar with the jovial Barry Evans character played by Shaun Williamson. You might have also seen him on "Celebrity Big Brother,"…
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