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Milasen - a drug developed for a single patient.
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease | NEJM: Summary Genome sequencing is often pivotal in the diagnosis of rare diseases, but many of these conditions lack specific treatments. We describe how molecular diagnosis of a rare, fatal neurodegenerative condition led to the...
Source: nejm.org
What’s the “Take Home” from Research on Dementia Trends?: Eric Larson and Kenneth Langa discuss whether the risk of
What’s the “Take Home” from Research on Dementia Trends?: Eric Larson and Kenneth Langa discuss whether the risk of dementia is increasing or decreasing over time. Eric B. Larson. Kenneth M. Langa. PLOS Medicine.
Source: plos.org
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