Aegerion
Homozygous familial hypercholesterolemia (HoFH)
CSL Behring
Primary and secondary immunodeficiencies(PID, SID); chronic inflammatory demyelinating polyneuropathy (CIDP); alpha 1 antitrypsin deficiency
EUSA
Neuroblastoma
Hormosan
Non-dystrophic myotonia (NDM)
Insmed
Infections with non-tuberculous mycobacteria (NTM)
Jerini
Hereditary angioedema (HAE)
Novartis Oncology
Myelofibrosis; pancreatic neuroendocrine tumor (pNET); chronic myeloid leukemia (CML)
PTC Therapeutics
Duchenne muscular dystrophy with nonsense mutation (nmDMD)
Sanofi Genzyme
M. Fabry, M. Pompe, M. Gaucher (lysosomal storage diseases); Acquired thrombotic thrombocytopenic purpura (aTTP)