Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
Leukodystrophy is the term used for a group of rare, progressive brain disorders which cause the brain's myelin sheath (the nerves protective covering) to either develop improperly or be destroyed…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
Meet Ruby, a beautiful 3 year old who was diagnosed with hypomyelination with atrophy of basal ganglia and cerebellum, or H-ABC, shortly after her birth. H-ABC is a type of leukodystrophy…
According to a story from abc.net.au, ever since Massimo Damiani was first diagnosed with a leukodystrophy, his parents, Sally and Stephen, have been committed to promoting research efforts and new…
Eva-Rose Sturgess is eighteen months old. She faces a disease for which there is no known cure: leukodystrophy. Her parents felt like their world came apart at the seams when…
A new treatment under development has produced promising results in patients with blood cancers and is now being tested in inherited metabolic disorders, reports Financial Buzz. Inherited metabolic disorders are…
You wouldn't know by looking at 5-year-old Grayson Ledbetter that he only had a few years of life left to live. According to The Washington Post, Grayson battles the rare,…
Losing a child has to be one of the most heart-wrenching experiences a family can encounter, yet losing a child when it could have been prevented is even more devastating.…
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.
