A US biopharmaceutical company in Austin, Texas, building a rare neurology franchise on one molecule, Aqneursa (levacetylleucine), approved for Niemann-Pick disease type C and ataxia-telangiectasia, with a third pivotal trial in CACNA1A-related disorders.
A US biopharmaceutical company in Austin, Texas, building a rare neurology franchise on one molecule, Aqneursa (levacetylleucine), approved for Niemann-Pick disease type C and ataxia-telangiectasia, with a third pivotal trial in CACNA1A-related disorders.
