List of Companies Profiled in the Report
AbbVie Inc.
Alexion Pharmaceuticals, Inc. (AstraZeneca)
Amgen Inc.
Amneal Pharmaceuticals LLC
Biogen Inc.
Bristol-Myers Squibb Company
Chiesi Farmaceutici S.p.A.
Dr. Reddy’s Laboratories Ltd.
F. Hoffmann-La Roche Ltd.
Johnson & Johnson Services, Inc.
Merck & Co Inc.
Novartis AG
Pfizer Inc.
Sanofi
Takeda Pharmaceutical Company Limited
Teva Pharmaceutical Industries Ltd.
Vertex Pharmaceuticals Incorporated

List of Other Companies Mentioned in the Report
2seventy bio, Inc.
Albireo
Beam Therapeutics
CANbridge Pharmaceuticals
Centogene N.V.
Century Therapeutics
ChemoCentryx
Dr. Falk Pharma GmbH
HUTCHMED
Immatics N.V.
Ipsen
LogicBio Therapeutics
MODAG GmbH
Novo Nordisk
Oryzon Genomics, S.A.
Parexel
Premier Research
Vertex Pharmaceuticals
ZEDIRA GmbH

List of Associations Mentioned in the Report
American Academy of Neurology (AAN)
Asia Pacific Alliance of Rare Disease Organisations (APARDO)
Brazilian Rare Disease Network (BRDN)
ERA-Net for Research Programmes on Rare Diseases (E-Rare)
European Federation of Neurological Societies (EFNA)
European Organisation for Rare Diseases (EURORDIS)
European Platform for Rare Disease Registries (EPIRARE)
European Union Committee of Experts on Rare Diseases (EUCERD)
International Coalition of Organizations Supporting Endocrine Patients (ICOSEP)
International Conference on Rare Diseases & Orphan Drugs (ICORD)
International Rare Diseases Research Consortium (IRDiRC)
National Organization for Rare Disorders (NORD)
Organization for Rare Diseases India (ORDI)
Rare Diseases International (RDI)
UK National Institute for Health and Care Excellence (NICE)