October 17, 2019
Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy
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September 17, 2019
A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
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September 15, 2019
EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
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September 15, 2019
An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis
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September 15, 2019
Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients
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September 14, 2019
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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September 14, 2019
Anti-CD19, Dual Co-stimulatory (4-1BB, CD3ζ) Chimeric Antigen Receptor T-cells in Patients With Relapsed/Refractory Aggressive Lymphoma or Acute Lymphoblastic Leukemia (ALL)
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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
Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases, Including (But Not Limited to) Those Who Have Completed the SCGAM-01 Trial
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September 14, 2019
Safety, Tolerability, and Efficacy of Cilofexor in Adults With Primary Sclerosing Cholangitis Without Cirrhosis
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September 14, 2019
Cholangioscopy in Primary Sclerosing Cholangitis (PSC)
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September 14, 2019
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
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