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

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.

Source: nature.com
Rescue of an orphan drug points to a new model for therapies for rare diseases - Vimarsana News

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.

Source: nature.com
Telethon Foundation Will Make and Distribute ADA-SCID Drug - Vimarsana News

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.

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

'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.

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

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