Categoria: Ricerca

Inizio del reclutamento per lo studio clinico ATTUNE

The ATTUNE trial is a Phase 1/2 clinical study sponsored by Ionis. It aims to evaluate ION440, an investigational Antisense Oligonucleotide (ASO) therapy designed to address the root cause of MECP2 Duplication Syndrome (MDS).

Inizia il reclutamento per HERO: studio clinico CRISPR cas13 per le SMD

The HERO clinical trial is a pioneering study evaluating HG204, a novel CRISPR Cas13 RNA-editing therapy, for MECP2 Duplication Syndrome (MDS). Sponsored by HuidaGene, this trial marks an important step in the search for a treatment for MDS. In November 2024, the first patient was successfully administered HG204, offering renewed hope to the MDS community.

ION440: IONIS porta la comunità MDS al livello successivo

Written by David and verified by Ionis Following Ionis’ recent announcement about the upcoming clinical trial, we aim to provide a clearer understanding of this trial. It is especially important for families considering enrolling their child to know what a clinical trial is, particularly in the context of rare diseases like MECP2 Duplication Syndrome (MDS). […]

HuidaGene: Verso la sperimentazione clinica

In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS). Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that […]

Huidagene: Una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina)

Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome, HG204 aims to regulate the […]