FDA Announces Big Push for Development of Gaucher Disease Treatment
Great news for our friends in the the Gaucher community! The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur…
Great news for our friends in the the Gaucher community! The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur…
Being told your child has a rare disease is devastating. Then imagine being told that there’s absolutely nothing you can do about it. That was the reality for these parents,…
Over ten years ago, four close friends founded a charity called Angie’s Hope. One of the founders, Angie Lee, has been living with spinal muscular atrophy (SMA) since she was…
I recently got together with a sweet friend, who has a six-month-old. It was the first time we'd actually talked face-to-face since her daughter was born, and the first time…
Baby Dylan had some sort of medical condition, as reported by The Global and Mail. His family was sure of it. He slept all the time and when he was…
You heard that right, Starbucks lovers! According to two different studies - one from the US and one from Europe - drinking anywhere from one to three cups of coffee…
Our wonderful partners at the Cystinosis Research Network (CRN) are providing the first series of live broadcasts from a Cystinosis conference! Simply make sure you're connected to the internet and…
A recent Canadian study published in Seminars in Arthritis and Rheumatism announced findings that psoriatic arthritis disease activity showed trends of stabilizing and even improving during pregnancy. Psoriatic arthritis is…
Every rare disease patient deserves an organization dedicated to working towards their specific condition. These organizations provide invaluable patient advocacy, information, support, and research funding for small communities that need…
You’ve been diagnosed with pulmonary arterial hypertension (PAH)… now what? Although there is currently no cure for pulmonary arterial hypertension, there are treatment options available and more are on the horizon.…
I recently read a report on the inspirational Lakiea Bailey, who has a rare disorder called sickle cell anemia. This disorder targets her red blood cells. Sickle cell diseases can…
According to Seacoast Online early this year, Cohl Capparelli was diagnosed with spinal muscular atrophy (SMA) about two years ago. SMA is one of the diseases that the Muscular Dystrophy Association…
What you’re about to read may sound like fiction. I promise you, it’s not. Two men from Tanzania had their boat capsize while fishing in the Indian Ocean a few…
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…
According to Today, Quinn Hoover is an 8-year-old boy who lives in Nebraska. His body doesn’t produce enough alkaline phosphatase enzyme, causing his bones to be extremely “soft." The condition…
It is easy to feel alone after being diagnosed with a rare disease. You are surrounded by others that can only empathize so much with what you are going through,…
According to The Newport Daily News, for Danielle and Kyle Ray, it was an all-too-common love story. They were school-aged sweethearts who married in their early 20s, shortly after having…