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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

Benefits seen in patients with and without a genetic diagnosis

Phase III trial falls short of efficacy target, but positive signal emerges in younger kids

If approved, this would be the first drug for Niemann-Pick disease type C

This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C

Two patients had rapid symptom improvement and reductions in malformation volume