A Call on the FDA for Timely Reviews of Life-Saving Therapies for Rare Diseases

The Need to Look Beyond Seizures When Treating DEEs

Taking On Big Competitors with an Oral Therapy to Treat Achondroplasia

Going Viral to Fight Brain Cancer

Changing the Playbook on Childhood Cancers

Sometimes the Hoofbeats Do Belong to Zebras

Breathing Easier with a Breakthrough Immunotherapy

Using AI Open Innovation to Tackle ALS

Recovering What’s Lost in Translation

Targeting Hunger Rather than Appetite in Prader-Willi Syndrome

A Rare-to-Common Business Model

Leading a Rare Youth Movement

Developing Gene Therapies that Work Across Mutations

Eyeing A Challenge to a Blockbuster

Like Mother, Like Child

Transforming from Grandmother to Mother to Advocate

Overcoming a Delivery Challenge of Retinal Gene Therapies

A Gene Editing First Augurs an Era of Bespoke Therapies

How One Patient Organization Drives Drug Development

Using CRISPR to Modulate Gene Expression