As Needham biotech struggles with FDA, patients with an ultra-rare disease are waiting

A drug made by a Needham-based biotech company called Stealth BioTherapeutics Inc. is a last-ditch source of hope for many patients. But it faces an uncertain future — as does its maker. For patient advocates, a back-and-forth between the FDA and a Needham biotech illustrates a major gap for patients with ultra-rare diseases.