Orah's Story: Refusing To Give Up with a USP7 Related Disease
My son Jacob seemed okay when he was born in September 2017, with minor feeding issues that went away. But as the months went on, some things began to disturb me.
Gene Transfer Therapy for Childhood Diseases: Everything You Need to Know
If you’ve spent any time reading up on or living life with a rare condition, chances are you’ve come across the terms “genetic disorder,” and “gene therapy.” But what exactly do they mean? And what differentiates them from other diseases and treatments?
How to Reduce Anxiety Before Medical Procedures or Treatments
Pre-procedure anxiety is a normal part of the medical process. In fact, up to 80 percent of surgical patients are reported to experience anxiety before operation.
Plasmapheresis: Procedure Information & Potential Complications
Plasmapheresis is a procedure used to filter out antibodies that are mistakenly attacking the body’s own tissues. Here’s what to know about the ins and outs of the procedure.
The Need for Clinical Studies in Rare Disease
Here’s a staggering statistic: only 5 percent of rare diseases have approved treatments. But that doesn’t mean there isn’t extensive work and research being done to find therapies, procedures, and eventually, cures for these conditions.
Tali's Story: Struggling with Epilepsy and Dravet Syndrome
On September 14, 2018 my healthy seven-month-old baby had her first seizure. It was scary. Really, really scary. It was hard to believe that this was really happening to us. After a lot of tests and tears, it became clear that this was not a one-time seizure.