- Corcept Therapeutics, Inc. – Cushing’s syndrome
Partner: Idis Sponsor: Corcept Therapeutics, Inc.
- Home
- /
- Access Program
- /
- Page 2
Access Programs
- Seattle Genetics, Inc. – Lymphoma, T-Cell, Cutaneous
Sponsor: Seattle Genetics, Inc.
- Vertex Pharmaceuticals Incorporated – Cystic Fibrosis
Sponsor: Vertex Pharmaceuticals Incorporated
- Cytori Therapeutics – Diffuse scleroderma
Partner: Idis Sponsor: Cytori Therapeutics
- M. Louise Markert – Complete DiGeorge Syndrome
Sponsor: M. Louise Markert - M. Louise Markert – DiGeorge syndrome
Sponsor: M. Louise Markert
- Children’s Hospital Medical Center, Cincinnati – Dravet Syndrome
Sponsor: Children's Hospital Medical Center, Cincinnati - M. Scott Perry – Dravet Syndrome
Sponsor: M. Scott Perry - M. Scott Perry – Dravet Syndrome
Sponsor: M. Scott Perry - University of Colorado, Denver – Dravet Syndrome
Sponsor: University of Colorado, Denver
- Marathon Pharmaceuticals, LLC. – Duchenne muscular dystrophy
Partner: Parexel International, LLC; Dohmen Life Science Services Sponsor: Marathon Pharmaceuticals, LLC. - PTC Therapeutics – Duchenne Muscular Dystrophy
Sponsor: PTC Therapeutics - PTC Therapeutics International Limited – Duchenne muscular dystrophy
Partner: TREAT-NMD; Cooperative International Neuromuscular Research Group (CINRG) Sponsor: PTC Therapeutics International Limited
- Meir Medical Center – Endometrial Cancer
Sponsor: Meir Medical Center
- Atara Biotherapeutics – Epstein-Barr Virus-Associated Viremia
Sponsor: Atara Biotherapeutics - Children’s Hospital of Philadelphia – Leukemia
Sponsor: Children's Hospital of Philadelphia - Hospital Ana Nery – Thromboembolism
Sponsor: Hospital Ana Nery - Johns Hopkins University – Anemia
Sponsor: Johns Hopkins University - Spectral Diagnostics (US) Inc. – Endotoxemia
Sponsor: Spectral Diagnostics (US) Inc. - Steven M. Frank, MD – Anemia
Sponsor: Steven M. Frank, MD - University of Miami – Anemia
Sponsor: University of Miami
- Eli Lilly and Company – Esophageal cancer
Sponsor: Eli Lilly and Company
- Novartis Pharmaceuticals – Essential thrombocytopenia
Sponsor: Novartis Pharmaceuticals
- Amicus Therapeutics – Fabry Disease
Sponsor: Amicus Therapeutics
- Amgen – Familial Melanoma
Sponsor: Amgen
- Baylor College of Medicine – Growth Failure
Sponsor: Baylor College of Medicine - Baylor College of Medicine – Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
Sponsor: Baylor College of Medicine - Children’s Hospital of Philadelphia – Bone Marrow Failure Syndrome
Sponsor: Children's Hospital of Philadelphia - Eli Lilly and Company – Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI)
Sponsor: Eli Lilly and Company - Gerard Vockley, MD, PhD – ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
Sponsor: Gerard Vockley, MD, PhD - Korea University Guro Hospital – Myocardial Infarction
Sponsor: Korea University Guro Hospital - Novartis Pharmaceuticals – Heart Failure With Reduced Ejection Fraction (HF-rEF)
Sponsor: Novartis Pharmaceuticals - University of California, San Francisco – Bone Marrow Failure
Sponsor: University of California, San Francisco - University of Pittsburgh – ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
Sponsor: University of Pittsburgh - Washington University School of Medicine – Erythrocytosis, Familial, 2
Sponsor: Washington University School of Medicine - Xiaofan Zhu – Fanconi Anemia
Sponsor: Xiaofan Zhu - Xiaofan Zhu – Fanconi Anemia
Sponsor: Xiaofan Zhu
- Kite Pharma, Inc. – Relapsed/Refractory Transformed Follicular Lymphoma
Sponsor: Kite Pharma, Inc. - Kite Pharma, Inc. – Relapsed/Refractory Transplant Ineligible Transformed Follicular Lymphoma
Sponsor: Kite Pharma, Inc. - Kite, A Gilead Company – Relapsed/Refractory Transformed Follicular Lymphoma
Sponsor: Kite, A Gilead Company
- Celldex Therapeutics – Recurrent GBM
Sponsor: Celldex Therapeutics - Center Trials & Treatment – Glioblastoma by Gene Expression Profile
Sponsor: Center Trials & Treatment - Center Trials & Treatment – Glioblastoma Multiforme
Sponsor: Center Trials & Treatment - Northwest Biotherapeutics – Glioblastoma
Sponsor: Northwest Biotherapeutics - Northwest Biotherapeutics – Glioblastoma Multiforme
Sponsor: Northwest Biotherapeutics
- Center Trials & Treatment – Glioma of Brain
Sponsor: Center Trials & Treatment - Oncoceutics, Inc. – Glioma
Sponsor: Oncoceutics, Inc.
- Juan Pascual – Glucose Transporter Type 1 Deficiency Syndrome
Sponsor: Juan Pascual - Juan Pascual – Glut1 Deficiency Syndrome
Sponsor: Juan Pascual
- University of California, San Francisco – Hemoglobinopathy
Sponsor: University of California, San Francisco
- Genentech, Inc. – Hemophilia A
Sponsor: Genentech, Inc. - GlaxoSmithKline – Systemic Lupus Erythematosus
Sponsor: GlaxoSmithKline - Joanne Kurtzberg, MD – Hypoxia Ischemia, Cerebral
Sponsor: Joanne Kurtzberg, MD - Paolo Caimi, MD – Hematopoietic/Lymphoid Cancer
Sponsor: Paolo Caimi, MD - Seoul National University Hospital – Chemical Burn
Sponsor: Seoul National University Hospital - Vitreous -Retina- Macula Consultants of New York – Retinal Hemangioma
Sponsor: Vitreous -Retina- Macula Consultants of New York - Xiaofan Zhu – Hematopoiesis Maintainance.
Sponsor: Xiaofan Zhu - Zimmer Biomet – Critical Limb Ischemia
Sponsor: Zimmer Biomet
- AbbVie – Hepatitis C Virus Infection
Sponsor: AbbVie
- H. Lee Moffitt Cancer Center and Research Institute – HepatoCellular Carcinoma
Sponsor: H. Lee Moffitt Cancer Center and Research Institute - Ming Zhao – Hepatocellular Carcinoma
Sponsor: Ming Zhao - Stanford University – Hepatocellular Carcinoma
Sponsor: Stanford University
- Alnylam Pharmaceuticals – Hereditary amyloidosis
Sponsor: Alnylam Pharmaceuticals
- Pharming Group N.V. – Hereditary angioedema
Partner: Idis Sponsor: Pharming Group N.V.
- Alnylam Pharmaceuticals – Amyloid Neuropathies, Familial
Sponsor: Alnylam Pharmaceuticals - Alnylam Pharmaceuticals – Amyloidosis, Hereditary, Transthyretin-Related
Sponsor: Alnylam Pharmaceuticals - Alnylam Pharmaceuticals – Familial Amyloid Polyneuropathies
Sponsor: Alnylam Pharmaceuticals - Alnylam Pharmaceuticals – Familial amyloid polyneuropathy
Sponsor: Alnylam Pharmaceuticals
- AbbVie – Non-Hodgkin’s Lymphoma
Sponsor: AbbVie - Molecular Templates, Inc. – Non-Hodgkin’s B-cell Lymphoma
Sponsor: Molecular Templates, Inc. - Seattle Genetics, Inc. – Lymphoma, Non-Hodgkin
Sponsor: Seattle Genetics, Inc.
- Impatients N.V. trading as myTomorrows – SR aGvHD
Sponsor: Impatients N.V. trading as myTomorrows
- Joanne Kurtzberg, MD – Hydrocephalus
Sponsor: Joanne Kurtzberg, MD
- University of Texas Southwestern Medical Center – Hypercalciuria- childhood idiopathic
Sponsor: University of Texas Southwestern Medical Center