William Maier

Biography

His promotion of a new pharmacoepidemiology, risk management, and research methodology—and his application of innovative technology to improve efficiency and accuracy of post-marketing surveillance studies—has proven invaluable to rare disease researchers.  Dr. Maier has been involved in drug development and regulatory negotiations for products indicated for the following rare / orphan indications:  Corneal cystine crystal deposits  Stem Cell Mobilisation in Healthy Donors  Severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS) following haematopoietic stem-cell transplantation (HSCT) therapy  Primary Immunodeficiency (PI) in adults. This included, but was not limited to, common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott- Aldrich syndrome, and severe combined immunodeficiencies  Cystic Fibrosis  Genetic variants of amyloid plaque development in Alzheimer’s Disease  Primary and secondary progressive Multiple Sclerosis  Crohn’s Disease and Ulcerative Colitis  Autosomal dominant polycystic kidney disease (ADPKD)  Levoleucovorin for Use In Combination Chemotherapy With 5-Fluorouracil In The Palliative Treatment Of Patients With Advanced Metastatic Colorectal Cancer: given orphan drug definition by FDA  He has also conducted research projects for the follow rare disease areas:   Cushing’s syndrome  Duchenne’s muscular dystrophy (DMD)  Progressive Multifocal Leukoencephalopathy (PML)  Niemann-Pick type C disease  Metastatic colorectal cancer (mCRC)  Malignant glioma  Short bowel syndrome (SBS)  Dupuytren’s contracture  Achondroplasia  Multiple myeloma  Transfusion-dependent anaemia due to myelodysplastic syndrome  Insulin-like growth factor-1 Deficiency (IGF-1)  Idiopathic pulmonary fibrosis (IPF)  Hepatic encephalopathy chronic hypoparathyroidism  N-Acetyl glutamate synthase deficiency  Vitreomacular traction lipoprotein lipase-deficiency (LPL)

Research Interest

drug development