📽️Film: ‘Rare Metabolic Diseases – Advancing Understanding, Improving Outcomes’

Watch now for FREE ⬇️
progress.org.uk/film-rare-me...

#MakeRareFair #MetabolicDisease #RareDisease #Health #Treatment #HealthSupport #NHS #Diagnosis #InheritedDisease #MetabolicSupportUK #DNA #Genetics

FDA clears first gene therapy for rare genetic disease WAS Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

#FondazioneTelethon ETS' Waskyra has become the first #genetherapy in the #US for patients with #WiskottAldrichsyndrome (WAS), a rare and life-threatening #inheriteddisease.

How a third parent's DNA can prevent an inherited disease An experimental technique that patches defective DNA with donated genetic material helped families at risk of passing rare illnesses to their children. (Image credit: ARTUR PLAWGO)

How a third parent's DNA can prevent an inherited disease #Science #HealthandMedicine #Genetics #InheritedDisease #HealthInnovation