BioMarin Pharmaceutical Iduronidase Talazoparib Amifampridine Phenylalanine ammonia-lyase PEGylation Metachromatic leukodystrophy David E. I. Pyott Mucopolysaccharidosis Prosensa Elosulfase alfa Valoctocogene roxaparvovec Elliott Investment Management Drisapersen Vosoritide Cerliponase alfa Tetrahydrobiopterin Lon Cardon Emil Kakkis Tetrahydrobiopterin deficiency Catalyst Pharmaceuticals Brand Institute Repligen Carlos Bonilla Pegvaliase PARP inhibitor Complete Response Letter BIOTECanada National Organization for Rare Disorders List of recombinant proteins List of California companies Pharmaceutical Research and Manufacturers of Americ…
ca Ultragenyx Phenylketonuria Buck Institute for Research on Aging List of pharmaceutical companies Alirocumab List of companies based in the San Francisco Bay Area Merck Group Nasdaq-100 Priority review Novato, California San Rafael, California Marin County, California Raymond C. Stevens Medication ALS Phyllis Gardner (clinical pharmacologist) Adeno-associated virus Regenerative medicine advanced therapy Leiden Bio Science Park Gene therapy Genpact List of largest biomedical companies by market capitalization List of drugs gr
anted breakthrough therapy designation List of S&P 400 companies Russell 1000 Index
Iduronidase
Talazoparib
Amifampridine
Phenylalanine ammonia-lyase
PEGylation
Metachromatic leukodystrophy
David E. I. Pyott
Mucopolysaccharidosis
Prosensa
Elosulfase alfa
Valoctocogene roxaparvovec
Elliott Investment Management
Drisapersen
Vosoritide
Cerliponase alfa
Tetrahydrobiopterin
Lon Cardon
Emil Kakkis
Tetrahydrobiopterin deficiency
Catalyst Pharmaceuticals
Brand Institute
Repligen
Carlos Bonilla
Pegvaliase
PARP inhibitor
Complete Response Letter
BIOTECanada
National Organization for Rare Disorders
List of recombinant proteins
List of California companies
Pharmaceutical Research and Manufacturers of America
Ultragenyx
Phenylketonuria
Buck Institute for Research on Aging
List of pharmaceutical companies
Alirocumab
List of companies based in the San Francisco Bay Area
Merck Group
Nasdaq-100