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Observational study points to genetic risk allele dose-response

Health news and commentary gathered by 麻豆传媒 staff

Single infusion also reduced total plasma kallikrein protein levels versus placebo

Long non-coding RNA implicated in human disease

Significant improvement beyond 100 weeks after single injection of mutation-agnostic MCO-010

Differences between delandistrogene moxeparvovec and placebo emerge in secondary outcomes

Finding on HLA-DRB1*01:03 supports need for more and earlier genetic studies

Panelists acknowledge difficulty generating stronger data in ultra-rare disease

10% of patients in trials leading to eli-cel's approval developed MDS or AML

Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease

Higher risk for ever use, increasing duration of use but no increase in BRCA2 carriers

FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C

Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder

Findings "shift the dial more in favor of an animal origin," said one expert