September 15, 2019
Tobramycin 300 mg Once-a-day (o.d.) Aerosol in Adults With Cystic Fibrosis
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September 15, 2019
ECMO for Lung Transplantation in Cystic Fibrosis Patients
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September 14, 2019
Diagnostic Accuracy of Preoperative Diagnostic Procedure in the Assessment of Lymph Node Metastases by NF-PanNENs
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September 14, 2019
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
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September 14, 2019
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
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September 14, 2019
Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
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September 14, 2019
Evaluating the Safety and Efficacy of Oral Lenvatinib in Medullary and Iodine-131 Refractory, Unresectable Differentiated Thyroid Cancers, Stratified by Histology
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January 30, 2019
Effect of EPA-FFA on Polypectomy in Familial Adenomatous Polyposis
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January 30, 2019
Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
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January 30, 2019
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
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January 30, 2019
Gene Therapy for Metachromatic Leukodystrophy (MLD)
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January 30, 2019
Derazantinib in Subjects With FGFR2 Gene Fusion Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
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