Aptose’s Tuspetinib Exceeds Expectations When Combined with Standard of Care Treatment Across Diverse Populations of Newly Diagnosed AML

Addition of TUS to VEN+AZA achieves CR/CRh responses in all (6/6, 100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, ...
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AAV-delivered PPT1 gene therapy shows promise for CLN1 disease

Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...