Charlie is one of the children taking part in the Natural Evolution Study (NHS) conducted by Ionis Pharmaceuticals in the United States. The study has
Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based
Le monde de la santé peut souvent sembler complexe, en particulier lorsqu’il s’agit de comprendre les différents systèmes et études
The world of healthcare can often seem complex, especially when it comes to understanding the various systems and studies that
On the 28th of October, members of our team: Ana (board member), Caroline (board member, vice president) and Dr. Gudrun
Exactly a year ago, the « Lasst uns MDS heilen – DupMECP2 » association came to life. The association’s story began with
The 10th of March 2023 sets a very important date in the rare disease community. Trofinetide has been approved by
MECP2 duplication syndrome (MDS) is a rare and progressive neurogenetic disorder affecting children. It is caused by a gene duplication
Matteo's parents, presidents of the association Lasst uns MDS heilen - Let's cure MDS, have launched a fundraising campaign on
The biannual family conference was held in Houston (Texas, USA) from September 23 to 24, 2022. It was organized by
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