Stoke Therapeutics: FDA Delay Doesn't Change Zorevunersen's Opportunity

Stoke Therapeutics advances TANGO ASO for Dravet, with Zorevunersen Phase 3 data showing seizure reduction and a delayed 2027 ...
Popular Science

New therapies offer hope for patients with rare genetic conditions

This article originally appeared in Knowable Magazine. The seizures started when Samantha Gundel was just four months old. By her first birthday, she was taking a cocktail of three different ...
Inverse

These Rare Genetic Diseases Are Finally Getting Attention From Drug Companies

The seizures started when Samantha Gundel was just four months old. By her first birthday, she was taking a cocktail of three different anticonvulsant medicines. A vicious cycle of recurrent pneumonia ...
GEN

Dosage Sensor Ensures Mammalian Biallelic Expression

Gene dosage mechanisms are required to maintain the expression of haploinsufficient genes properly, which, if misregulated, can lead to many developmental disorders. For the first time, researchers ...
Nature

A haploinsufficiency restoration strategy corrects neurobehavioral deficits in Nf1+/– mice

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...