Common-sense reform for the review of ultra-rare disease therapies by the Food and Drug Administration sounded like a dream ...

Common-sense reform for the review of ultra-rare disease therapies by the Food and Drug Administration sounded like a dream come true for families like mine who are living with incurable, lethal ...

Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving ...

Last year, the patient community rallied to prepare for the FDA Advisory Committee meeting held to discuss Stealth Therapeutics’ drug elamipretide for the ultra-rare disorder Barth syndrome. The ...

When women ask for increased variable compensation, it sends a strong message: They’re betting on themselves. That’s music to ...

The star shares James, who is non-verbal, with his ex-partner, Kim Bordenave. He has Angelman syndrome - a rare disorder that affects the nervous system and causes developmental delays among other ...

And do you also have a tummy ache? If so, you might just have “good girl syndrome” — so says Diane Lange, a therapist and “positive living expert” whose most recent book, “Worthy ...

The disorder is called CerTra syndrome, and it is not hereditary. People with CerTra syndrome have developmental delays, seizures, traits similar to autism, visual and hearing differences ...

Before collapsing, he recently learned that he had the gene for something called Brugada syndrome. Years earlier, his brother was diagnosed with it after collapsing several times while playing ...