How One Woman’s Journey with a Rare Disease Motivated Her to Advocate for Herself and Others
Managing the unpredictable symptoms of indolent systemic mastocytosis prompted a search for answers and community.
Long read sequencing reveals more genetic information while cutting time and cost of rare disease diagnoses
One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid ...
Father's journey to support son with rare condition
Sky News host Paul Murray sits down with Stu Place who discusses his four-year-old son Will, who suffers from a rare ...
What Is Piriformis Syndrome? All About Andie MacDowell's 'Rare' Diagnosis
Piriformis syndrome is a rare neuromuscular condition that happens when the piriformis muscle in the butt presses on the ...
News Medical3d
What is an Orphan Drug? A Guide to Rare Disease Therapies
However, certain diseases and medical conditions continue to receive less funding and attention compared to others. Patients with rare medical conditions and diseases are often part of a ...
Pharm Exec8d
Casey McPherson Shares His Journey With Rare Drug Development and Chrysalis Genetics
Casey McPherson, Founder and CEO of Chrysalis Genetics, talks about his daughter's rare genetic condition, and how it led him to antisense oligonucleotides (ASOs) and therapeutic development for rare ...