Familial adenomatous polyposis (FAP) is caused by mutations in the APC gene and 2 different, or biallelic mutations, in the MUTYH gene. However, not all patients with colorectal polyposis are found to ...
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, ...
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 ...
Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5, an investigational gene therapy for Leber ...
Outcomes of Radium-223 and Stereotactic Ablative Radiotherapy Versus Stereotactic Ablative Radiotherapy for Oligometastatic Prostate Cancers: The RAVENS Phase II Randomized Trial We analyzed a cohort ...
Several rare immune disorders are caused by mutations in the patient's DNA. The University of Surrey and its collaborators found that targeting the mutated pathways with new drugs offers new hope for ...
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