Rare Genetic Diseases

Rare Genetic Diseases Programs

Lymphatic Malformations

  • AVTX-006, a dual mTOR inhibitor (a class of drugs that inhibit the mammalian target of rapamycin) in development for the treatment of lymphatic malformations Learn More >

Congenital Disorders of Glycosylation (CDG)

  • AVTX-801, a therapeutic dose of monosaccharide therapy for PGM1-CDG, an ultra-rare CDG Learn More >
  • AVTX-802, a therapeutic dose of monosaccharide therapy for MPI-CDG, an ultra-rare CDG Learn More >
  • AVTX-803, a therapeutic dose of monosaccharide therapy for LAD II, an ultra-rare CDG Learn More >