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Patient Stories

Guest Blog: A Parent Advocate & Author Discusses Courage

By David Cry, Guest Blogger

David CryCourage. Strength in the face of pain or grief. A tenet I have experienced on many occasions as the lead of… Read More

The Fight for a Cure for Duchenne

When Robert and Theresa Capolongo were preparing to be parents, many words were thrown around by their friends and family about the joys of having children. “Fulfilling.” “Life-changing.” “Beautiful.” These were, of course, balanced… Read More

Zan’s Journey: Diagnosed with SMS

When a psychiatrist diagnosed Robyn Fell’s toddler on the Autism spectrum, she was in shock.   “It’s not that I was in denial,” she explained, “my son, Zan, did have many unusual behaviors.”   A personality… Read More

The Will to Live and the Strength for a Cure

The day Joshua Frase was born, his parents Alison and Paul, were given grave news about their newborn’s life – he most likely would not survive that day. His weakened muscles could not function… Read More

Saving Eliza

I’m honored to be speaking at the NORD Breakthrough Summit on Rare Diseases and Orphan Products in October.   Social media, as I continue to learn more every day, is an always changing, rapidly moving,… Read More