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,…
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