First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.

A series of basic research insights around the cell death process called ferroptosis could form the underpinnings of a new class of treatments aimed at drug resistant tumors.

Newron Pharmaceuticals will file its investigational drug sarizotan in 2018 for respiratory issues in patients with Rett syndrome, setting it up to be the first therapy specifically indicated for this rare genetic disease. CEO Stefan Weber spoke to Scrip about Newron's commercial strategy for sarizotan and broader prospects for the evolving drug developer.

Emerging Company Profile: Decibel Therapeutics, a Boston-based company focused on developing therapies for hearing loss, started adding its voice in an emerging development space in 2015, with the aim of becoming "the premier hearing therapies company." With ongoing research and preclinical candidates for hearing loss, Decibel is looking to secure a beneficial collaboration that will bump up its profile and give it the funding it needs to move forward.