This Rare Disease Day is an opportunity to raise awareness of the challenges those living with a rare disease face, and why there’s reason to hope, thanks to advances in science that could potentially change the lives of millions of patients.
Skyclarys, a Reata Pharmaceuticals drug, is now approved for treating the rare neuromuscular disorder Friedreich’s ataxia. The decision makes the capsule the first therapy for a disease that’s rarer than either Duchenne muscular dystrophy or spinal muscular atrophy.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
While Covid has made many aspects of living with a rare disease more difficult, it has inspired policies and techniques that could help rare disease research progress more smoothly both during and after the pandemic.
Of the 30 million Americans affected by rare diseases, more than half are children. It's a tough population to treat, but we can start with a simple two-part question: How is the disease affecting the child’s life and what can we do to make it better?