Fondazione Telethon advances national Duchenne registry

01 / 04

A nationwide network of 16 Italian neuromuscular centers and over 1,200 patients to support research and new therapies.

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Telethon Undiagnosed Disease Program

02 / 04

Over eight years, nearly one in two children received a molecular diagnosis

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Collaboration between Cytiva and SR-TIGET

03 / 04

Objective: to develop next-generation genomic medicine platforms

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Announced FDA approval of Waskyra™

04 / 04

Fondazione Telethon announces that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott-Aldrich syndrome (WAS).

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Our numbers

Since 1990 we fund research on rare genetic conditions

741 Millions of euros invested
1.916 Researchers
3.118 Projects funded

What we do

Our mission

We are an Italian non-profit organization committed to advancing research on rare and complex genetic diseases. For over 35 years, we have supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.

Institutes

We established two Institutes in Italy, now internationally recognized as centers of excellence: The San Raffaele Telethon Institute For Gene Therapy in Milan and Telethon Institute of Genetics and Medicine in Pozzuoli (NA).

Research initiatives

We drive excellence and innovation in research on rare genetic diseases, building a pipeline that spans from basic science to clinical trials and approved therapies.

The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS

Storie e News

Fondazione Telethon have obtained marketing authorisation for a gene therapy thanks to a sustainability model that integrates research with industry, patients and supporters. Read the full interview with Celeste Scotti, R&D Director at Fondazione Telethon.

News

Read all the news

29.05.26

Fondazione Telethon advances national Duchenne registry for research and regulation

A nationwide network of 16 Italian neuromuscular centers and over 1,200 patients to support research and new therapies.

From Telethon Foundation

19.05.26

SR-Tiget’s Development Facilities: where gene therapy takes shape

How SR-Tiget’s development facilities are key to turn research into advanced therapies, and why they represent a new professional frontier.

From Research

08.05.26

Lysosome Biology: From “Suicide Bag” to Metabolic Hub

Tigem research reveals the lysosome as a metabolic hub via TFEB and mTOR, enabling new treatments for lysosomal diseases, neurodegeneration and cancer.

From Research

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