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Gene therapies for rare diseases are under threat. Scientists hope to save them.

As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them. As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them. ....

City Of , United Kingdom , United States , France General , South San Francisco , Luigi Naldini , Simone Boselli , Eric Piermont , Los Angeles , Donald Kohn , Francesca Pasinelli , Barbara Ballard , Drug Administration , Telethon Foundation , Angels For Life Foundation , Us National Center , San Raffaele Telethon Institute For Gene Therapy , Translational Sciences , California Institute Of Regenerative Medicine , Office Of Health Economics , University Of California , Advancing Translational Sciences , Life Foundation , San Raffaele Telethon Institute , Gene Therapy , Orchard Therapeutics ,

Rescue of an orphan drug points to a new model for therapies for rare diseases

In a world-first, the Italian research charity Telethon will manufacture and distribute a gene therapy for an inherited immunodeficiency that was dropped by the industry. In a world-first, the Italian research charity Telethon will manufacture and distribute a gene therapy for an inherited immunodeficiency that was dropped by the industry. ....

City Of , United Kingdom , Francesca Pasinelli , Piemonte Orientale , Andrew Brookes Getty , Armando Genazzani , European Medicines Agency , European Union , National Health Service For Italian , Telethon Foundation , Italian Society Of Pharmacology , Telethon Institute For Gene Therapy , Orchard Therapeutics , San Raffaele Telethon Institute , Gene Therapy , Del Piemonte Orientale , Italian Society , San Raffaele Campus , Telethon Institute , San Raffaele , National Health Service , Wiskott Aldrich Syndrome , Great Ormond Street Hospital ,

Telethon Foundation Will Make and Distribute ADA-SCID Drug

The initiative will ensure the availability of a drug that otherwise would have been abandoned as unprofitable. ....

Alessandro Aiuti , Francesca Pasinelli , Foundation Steps , National Health Service For Italian , Scientific Institute For Research , Telethon Foundation , San Raffaele Telethon Institute For Gene Therapy , European Union , European Medicines Agency , Pediatric Immunohematology Unit , Scientific Institute , San Raffaele Hospital , San Raffaele Telethon Institute , Gene Therapy , Orchard Therapeutics , San Raffaele , National Health Service , Primary Immunodeficiency , Primary Immunodeficiencies , Severe Combined Immunodeficiency , Transplantation Stem Cell , Ransplantation Hematopoietic Stem Cell , Hematopoietic Stem Cell Transplantation , Stem Cell Transplantation , Stem Cell Therapy , Herapy Stem Cell ,

'Bubble Boy' Disease Has a Cure, but Drug Companies Won't Provide It

Jakob, who was born with a rare illness, is 2 years old. But what's keeping him isolated from the world isn't his disease it's the marketplace. ....

United States , Stanford University , United Kingdom , Matthew Porteus , Donald Kohn , Alessandro Aiuti , Paola Andrea Fern , Manny Lichtman , John Travolta , Ceos Andrea , Maria Grazia Roncarolo , David Vetter , Emma Walmsley , Jakob Kamil Guziak , Peter Marks , Ethan Perlstein , Francesca Pasinelli , Soto Abdul Rahim , Jennifer Doudna , Jude Children Research Hospital , Division Of Rare Diseases Research Innovation , National Institutes Of Health , Drug Administration , Telethon Foundation , Big Pharma , Texas Children ,

IAMA Therapeutics Partners with the Italian Institute of Technology to Embark on Landmark Research on Neuroscience Drug Discovery

IAMA Therapeutics, a Series A preclinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel medicines to make a difference in the lives of individuals ....

Matteo Bonfanti , Kostenloser Wertpapierhandel , Francesca Pasinelli , Andreap Malizia , Fondazione Telethon The Foundation , Foreign Press Office , Ministry Of Economic , Italiano Di Tecnologia Italian Institute Of Technology , Technology Transfer At Istituto Italiano Di Tecnologia , Italian Ministry Of Education , Telethon Foundation , Istituto Italiano , Italian Institute , Technology Transfer , Fondazione Telethon , General Manager , Italian Ministry , Press Office ,