- Cook – Abdominal aortic aneurysm
Sponsor: Cook - Cordis Corporation – Abdominal Aortic Aneurysm
Sponsor: Cordis Corporation
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Access Programs
- Novartis Pharmaceuticals – Acromegaly
Sponsor: Novartis Pharmaceuticals
- Amgen – Acute lymphoblastic leukemia
Partner: Amgen Sponsor: Amgen - ERYtech Pharma – Acute lymphoblastic leukemia
Sponsor: ERYtech Pharma - ERYtech Pharma – Acute Lymphoblastic Leukemia
Sponsor: ERYtech Pharma - Novartis Pharmaceuticals – Acute Lymphoblastic Leukemia
Sponsor: Novartis Pharmaceuticals - University of California, San Francisco – Acute lymphoblastic leukemia
Sponsor: University of California, San Francisco - University of California, San Francisco – Acute Lymphoblastic Leukemia
Sponsor: University of California, San Francisco
- Celator Pharmaceuticals – Secondary AML
Sponsor: Celator Pharmaceuticals - Children’s Hospital Los Angeles – Acute myeloid leukemia
Sponsor: Children's Hospital Los Angeles - Emory University – Acute myeloid leukemia
Sponsor: Emory University - Jazz Pharmaceuticals – Secondary AML
Sponsor: Jazz Pharmaceuticals - University of California, San Francisco – Acute myeloid leukemia
Sponsor: University of California, San Francisco
- Seattle Genetics, Inc. – Lymphoma, Large-Cell, Anaplastic
Sponsor: Seattle Genetics, Inc.
- Ariad Pharmaceuticals – Advanced Malignancies
Sponsor: Ariad Pharmaceuticals - Eisai Inc. – Seizure Disorder Generalized Tonic Clonic
Sponsor: Eisai Inc. - European Institute for Evidence Based Osteopathic Medicine – The Focus of the Study is to Provide a Guideline on Osteopathic Approach in Treating Hospitalized Newborns.
Sponsor: European Institute for Evidence Based Osteopathic Medicine - Joanne Kurtzberg, MD – Hematologic Malignancies
Sponsor: Joanne Kurtzberg, MD - Kochi University – Both Benign and Malignant Liver Mass
Sponsor: Kochi University - M.D. Anderson Cancer Center – Malignant Neoplasms of Digestive Organs
Sponsor: M.D. Anderson Cancer Center - M.D. Anderson Cancer Center – Malignant Neoplasms of Eye Brain and Other Parts of Central Nervous System
Sponsor: M.D. Anderson Cancer Center - SynCore Biotechnology Co., Ltd. – Malignant Solid Tumour
Sponsor: SynCore Biotechnology Co., Ltd. - University of California, San Francisco – Anemia Due to Intrinsic Red Cell Abnormality
Sponsor: University of California, San Francisco - Xiaofan Zhu – Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.
Sponsor: Xiaofan Zhu
- Boehringer Ingelheim – Adenocarcinoma of lung
Sponsor: Boehringer Ingelheim
- Eisai Inc. – Partial Onset Seizures
Sponsor: Eisai Inc.
- University of Colorado, Denver – Aggressive Fibromatosis
Sponsor: University of Colorado, Denver
- Eli Lilly and Company – Aicardi-Goutières Syndrome (AGS)
Sponsor: Eli Lilly and Company
- Fudan University – Triple Negative Local Advanced Breast Cancer
Sponsor: Fudan University - University of Roma La Sapienza – Locally Advanced Pancreatic Cancer
Sponsor: University of Roma La Sapienza
- EyeTechCare – Refractory Open Angle Glaucoma
Sponsor: EyeTechCare - Joanne Kurtzberg, MD – Autism
Sponsor: Joanne Kurtzberg, MD - Joanne Kurtzberg, MD – Autism Spectrum Disorder
Sponsor: Joanne Kurtzberg, MD
- ArQule – Growth Disorders
Sponsor: ArQule - Baylor College of Medicine – Hyperinsulinemic Hypoglycemia, Persistent
Sponsor: Baylor College of Medicine - Christine Hunter – Hyperkinetic Movement Disorders
Sponsor: Christine Hunter - Gerard Vockley, MD, PhD – Glycogen Storage Disorders
Sponsor: Gerard Vockley, MD, PhD - Ignyta, Inc. – Cancers With NTRK, ROS1, or ALK Gene Fusions
Sponsor: Ignyta, Inc. - Ignyta, Inc. – Cancers With RET Alterations
Sponsor: Ignyta, Inc. - Impatients N.V. trading as myTomorrows – Acute Graft-Versus-Host Reaction Following Bone Marrow Transplant
Sponsor: Impatients N.V. trading as myTomorrows - Impatients N.V. trading as myTomorrows – Acute-graft-versus-host Disease
Sponsor: Impatients N.V. trading as myTomorrows - Impatients N.V. trading as myTomorrows – Graft-versus-host-disease
Sponsor: Impatients N.V. trading as myTomorrows - Impatients N.V. trading as myTomorrows – Steroid Refractory Acute Graft Versus Host Disease
Sponsor: Impatients N.V. trading as myTomorrows - Incyte Corporation – Graft-versus-host Disease (GVHD)
Sponsor: Incyte Corporation - Joanne Kurtzberg, MD – Inborn Errors of Metabolism Disorders
Sponsor: Joanne Kurtzberg, MD - Jonsson Comprehensive Cancer Center – Cancers Expressing Somatostatin Receptors
Sponsor: Jonsson Comprehensive Cancer Center - M. Louise Markert – Low T Cell Numbers
Sponsor: M. Louise Markert - University of California, San Francisco – Leukocyte Disorders
Sponsor: University of California, San Francisco - University of Pittsburgh – Glycogen Storage Disorders
Sponsor: University of Pittsburgh
- Alnylam Pharmaceuticals – Amyloid Neuropathies
Sponsor: Alnylam Pharmaceuticals
- British Columbia Cancer Agency – Anotia facial palsy cardiac defect
Sponsor: British Columbia Cancer Agency
- Joanne Kurtzberg, MD – Apraxia of Speech
Sponsor: Joanne Kurtzberg, MD
- Tessa L Marburger – Congenital Myasthenia (CM)
Sponsor: Tessa L Marburger
- University of California, San Francisco – B-cell lymphomas
Sponsor: University of California, San Francisco
- Gerard Vockley, MD, PhD – Barth Syndrome
Sponsor: Gerard Vockley, MD, PhD - University of Pittsburgh – Barth Syndrome
Sponsor: University of Pittsburgh
- Children’s Hospital of Philadelphia – Beckwith-Wiedemann Syndrome
Sponsor: Children's Hospital of Philadelphia
- University of California, San Francisco – Bone Marrow failure syndromes
Sponsor: University of California, San Francisco
- Andrei Iagaru – Carcinoid tumor
Sponsor: Andrei Iagaru - British Columbia Cancer Agency – Carcinoid Tumor
Sponsor: British Columbia Cancer Agency - Johannes Czernin – Carcinoid tumor
Sponsor: Johannes Czernin
- Advanced Accelerator Applications – Midgut Carcinoid Tumor
Sponsor: Advanced Accelerator Applications - Andrei Iagaru – Carcinoid Tumors
Sponsor: Andrei Iagaru - Ariad Pharmaceuticals – Carcinoma
Sponsor: Ariad Pharmaceuticals - Jonsson Comprehensive Cancer Center – Carcinoid Cancer
Sponsor: Jonsson Comprehensive Cancer Center - Jonsson Comprehensive Cancer Center – Urothelial Carcinoma Ureteral Location
Sponsor: Jonsson Comprehensive Cancer Center - MacroGenics – HER2-positive Carcinoma
Sponsor: MacroGenics - Pfizer – Carcinoma, Non-Small-Cell Lung
Sponsor: Pfizer
- Baylor College of Medicine – Cholestasis
Sponsor: Baylor College of Medicine - Catherine Cibulskis, MD – Cholestasis
Sponsor: Catherine Cibulskis, MD - Children’s Hospital of Philadelphia – Cholestasis
Sponsor: Children's Hospital of Philadelphia - Geisinger Clinic – Cholestasis
Sponsor: Geisinger Clinic - Georgetown University – Cholestasis
Sponsor: Georgetown University - Georgetown University – Cholestasis of Parenteral Nutrition
Sponsor: Georgetown University - Rush University Medical Center – Cholestasis
Sponsor: Rush University Medical Center
- Emory University – Chronic myeloid leukemia
Sponsor: Emory University - Emory University – Chronic Myeloid Leukemia
Sponsor: Emory University - Pfizer – Chronic myeloid leukemia
Sponsor: Pfizer - Pusan National University Hospital – Chronic myeloid leukemia
Sponsor: Pusan National University Hospital - Pusan National University Hospital – Chronic Myeloid Leukemia
Sponsor: Pusan National University Hospital - University of California, San Francisco – Chronic myeloid leukemia
Sponsor: University of California, San Francisco - University of California, San Francisco – Chronic Myeloid Leukemia
Sponsor: University of California, San Francisco
- Baylor College of Medicine – CMV antenatal infection
Sponsor: Baylor College of Medicine
- Joanne Kurtzberg, MD – Common variable immunodeficiency
Sponsor: Joanne Kurtzberg, MD
- Catalyst Pharmaceutical Partners, Inc – Congenital myasthenic syndrome with episodic apnea
Sponsor: Catalyst Pharmaceutical Partners, Inc - Tessa L Marburger – Congenital myasthenic syndrome with episodic apnea
Sponsor: Tessa L Marburger
- ProMetic BioTherapeutics, Inc – Congenital Plasminogen Deficiency
Sponsor: ProMetic BioTherapeutics, Inc
- Impatients N.V. trading as myTomorrows – Alpha 1-Antitrypsin Deficiency
Sponsor: Impatients N.V. trading as myTomorrows