#MCT8 deficiency or #Allan_Herndon_Dudley syndrome is a rare disorder of #thyroid hormone transport, leading to severe neurodevelopmental delay. See our newest paper for monitoring recommendations.

https://pubmed.ncbi.nlm.nih.gov/41926547/
https://doi.org/10.1159/000551857
https://www.livivo.de/doc/M41926547

#MCT8 Deficiency, or Allan-Herndon-Dudley syndrome, is a rare severe disorder of #thyroid hormone transport. In our newest paper, we review the state of the art on pathophysiology, diagnosis & treatment.

https://pubmed.ncbi.nlm.nih.gov/41508830/
https://doi.org/10.1210/clinem/dgaf707
https://www.livivo.de/doc/M41508830

Egetis initiates rolling NDA for Emcitate

Egetis Therapeutics has started a rolling New Drug Application (NDA) to the US FDA for Emcitate (tiratricol) for the treatment of MCT8 deficiency. This follows a pre-NDA meeting on October 21 where the FDA confirmed eligibility based on available data and granted the rolling review process.

#biotech #FDA #MCT8 #Egetis