Rett In The News
Increasing expression of the gene that is faulty in Rett syndrome lowers repetitive behaviors in a mouse model of a different autism-linked condition called Pitt-Hopkins syndrome, a new unpublished study demonstrates.
Taysha Gene Therapies
Taysha Receives Orphan Drug Designation from the European Commission for TSHA-102 for the Treatment of Rett Syndrome
Taysha Gene Therapies today announced that it has been granted orphan drug designation from the European Commission for TSHA-102, an AAV9-based gene replacement therapy in development for Rett syndrome. An orphan designation allows a pharmaceutical company to benefit from incentives such as ...
Two years after spinning out of CRISPR pioneer Prashant Mali’s lab, Shape Therapeutics has a lot more cash and a slightly new mission. They have lead programs in Rett syndrome and Parkinson’s.
In their own words - members of RSRT’s newly formed Translational Medicine Advisory Board share their reasons for optimism
Rett Syndrome is considered a rare disease; yet rare diseases, as a whole, are not rare—they ...