Therapy areas


Inborn errors of metabolism / enzyme deficiencies

Numerous chemical processes are involved in the metabolism of food nutrients and other substances we ingest. In many, if not most, of these processes enzymes are involved and catalyze the chemical reactions. These enzymes are produced by the human cells based on the genetic code in the DNA.  If a person is born with a genetic alteration  which produces a defect enzyme or no enzyme at all, this might have consequences which manifest as different  symptoms and diseases, often already in infancy or early childhood. There are many enzymes and chemical processes in the body and consequently there are many processes which can be affected leading to multiple types of diseases. Medical Need is working in some disease areas with medical information,education and development of treatments.

  • Congenital Sucrase-Isomaltase Deficiency (CSID)
  • Urea Cycle Disorders (UCD)
  • Niemann-Pick Type C


  • Progressive Multiple Sclerosis
  • Vertigo of various origin
  • Oncology/hematology
  • Hematopoietic Stem Cell Transplantation / solid tumors

Infectious & Inflammatory diseases

  • Multi-Drug Resistant Tuberculosis
  • Chronic remitting pouchitis