Kumail was diagnosed with spinal muscular atrophy (SMA) and started Spinraza in an international trial. As The Herald Sun reports, he began the treatment at six weeks old before he…
Could crizanlizumab be the next treatment to prevent pain in those with sickle cell disease (SCD)? Is this the medicine you've been hoping for? Sickle cell disease (SCD) is an…
There's some new information available about Parkinson's disease, and researchers are really excited about it. Earlier this month, researchers found that visual system changes in recently diagnosed Parkinson's patients can…
β-thalassemia major is a rare blood condition that necessitates a lifetime of blood transfusions. Despite advancements in treatments, the perfect cure has yet to be found. Bluebird Bio, Inc. is…
No two patients a doctor sees will ever be exactly alike. They all will have different symptoms, allergies, and conditions, in addition to different backgrounds, personalities, and identities. While you…
A new study detailed in Nature, involving synthetically replicating certain cell activity has been hailed as a major breakthrough in developing therapies for many neurological disease – Parkinson’s chiefly among…
According to SBS Australia, Duncan Whitcombe and his son Bailey are both living with a rare neurological condition: Tourette syndrome. Duncan has had Tourette's since he was 10. At that…
According to Port City Daily, 21-year-old Haley Swanson has been living with dermatomyositis for 10 years. She was diagnosed at age 11 with the autoimmune condition, which means that her…
You have probably seen them, or at least heard of them. In fact, I’d go so far as to say you’ve seen them within the past week or two. 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.
