Scientists at St. Jude Children's Research Hospital have found that they can reverse the effects of HNRNPH2-related ...

Patients treated with high-dose BIIB080 saw positive trends on the global Clinical Dementia Rating Sum of Boxes, Mini-Mental State Exam cognitive scales and Functional Activities Questionnaire at week ...

Although the concept of using synthetic oligonucleotides to modulate RNA function was described as early as 1978, 1 its realization in the form of drugs approved by the Food and Drug Administration ...

The right approaches are demonstrating that it is possible to breach the defensively designed blood-brain barrier despite its ...

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...

Trace Neuroscience, founded in January, has launched with $101 million raised in a series A financing round to advance its antisense oligonucleotide therapy (ASO) treatment for amyotrophic lateral ...