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)