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Kategori: Forskning

Start af rekruttering til det kliniske ATTUNE-forsøg

ATTUNE-forsøget er et klinisk fase 1/2-studie sponsoreret af Ionis. Det har til formål at evaluere ION440, en undersøgende antisense-oligonukleotid (ASO)-behandling, der er designet til at adressere den grundlæggende årsag til MECP2 Duplication Syndrome (MDS).

Rekruttering starter til HERO: CRISPR cas13-klinisk forsøg med MDS

Det kliniske HERO-forsøg er et banebrydende studie, der evaluerer HG204, en ny CRISPR Cas13 RNA-redigeringsterapi, til MECP2 Duplication Syndrome (MDS). Forsøget, der er sponsoreret af HuidaGene, markerer et vigtigt skridt i jagten på en behandling af MDS. I november 2024 fik den første patient HG204 med succes, hvilket giver fornyet håb til MDS-samfundet.

ION440: IONIS bringer MDS-fællesskabet op på næste niveau

ionis attune

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: På vej mod kliniske forsøg

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: En lovende CRISPR-CAS13-teknologi fra Shanghai (Kina)

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 […]