genetic disease

Dehydrodolichyl diphosphate synthase (DHDSS)-related disease has no available treatments. The new work could finally change that with  nicotinamide mononucleotide (NMN).

Oral FGFR3-targeted therapies are emerging as a potential step forward, offering more precise, convenient treatment options for children.

Paolo Martini leads the biotech company’s rare disease research program with the hope of developing the very first treatments for genetic metabolic disorders.

Discover causes, treatments, and patient-driven solutions for chronic hiccups, a rare and mysterious condition.

A one-time gene therapy aims to stop Huntington's disease at its source. But first, it has to survive clinical trials.

A collaboration of nephrologists and biochemists created the first structure of “megalin,” a critical protein in kidney and brain disorders.

LIVE May 12th, 2022 at 2:30 PM
Discover the most current cell therapy research and the challenges of translating them into the clinic.