Researchers have demonstrated that heteroduplex oligonucleotide drugs conjugated with cholesterol efficiently cross the blood-brain barrier [...]
In this paper a British researcher argues that some human reproductive genome editing interventions can [...]
Cancer is one of the most feared diagnoses, and any treatment that increases the chance [...]
This phase 3 randomized, observer-blinded, placebo-controlled trial was conducted at 99 centers across the United [...]
CAR T-cell therapy has garnered significant excitement due to its success for hematological malignancies in [...]
Mitochondrial diseases are predominantly caused by mutations of mitochondrial or nuclear DNA, resulting in multisystem [...]
This paper addresses the question of what a reasonable price for an orphan drug is. [...]
Therapeutic RNA molecules possess high potential for treating medical conditions if they can successfully reach [...]
There are challenges in conducting a budget impact analysis (BIA) for rare disorders. Through this [...]
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