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A Phase 2, Two-Stage, Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of Rls-0071 in Newborns with Moderate ...
Hypoxic-ischemic encephalopathy (HIE) affects approximately 4,000 to 12,000 persons annually in the United States. Mortality from HIE has been reported up to 60%, with at least 25% of survivors left ...
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy ...
To evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients.THIS STUDY IS ENROLLING BY INVITATION ONLY - The study will invite participants with DM1 who completed ...
Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of Dyne-101 Administered to Participants with Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).THIS STUDY ...
NF119: a Phase 2, Open-Label, Single Arm, Non-Controlled, Single-Stage Study to Evaluate the Safety and Effects of HLX-1502 in Patients with Plexiform Neurofibroma and Neurofibromatosis ...
The trial will be an open label, single arm, phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 ...
Kysa-6: a Phase 2/3, Open-Label, Randomized, Controlled, Multicenter Study of Kyv-101, an Autologous Fully Human Anti-Cd19 Chimeric Antigen Receptor T-Cell (Cd19 Car T) Therapy, Versus ...
Myasthenia gravis (MG) is a chronic autoimmune disease that affects the neuromuscular junction and is characterized by muscle weakness. B cells play a role in MG, and the disease is ...
1011003300 National Cell Repository for Alzheimer's Disease
Does more than one member of your family have Alzheimer’s disease or serious memory loss? Your family can be an essential part of Alzheimer's disease research by participating in the ...
1011003300 National Cell Repository for Alzheimer's Disease
Does more than one member of your family have Alzheimer’s disease or serious memory loss? Your family can be an essential part of Alzheimer's disease research by participating in the ...
1011003338 Indiana Alzheimer Disease Core Center
The purpose of this program is to identify, evaluate, study, and follow persons who have Alzheimer's disease, those who have other disorders of memory and thinking, and older individuals who ...
1011003338 Indiana Alzheimer Disease Core Center
The purpose of this program is to identify, evaluate, study, and follow persons who have Alzheimer's disease, those who have other disorders of memory and thinking, and older individuals who ...
A Double-Blind, Randomized, Placebo-Controlled, Multicenter, Outpatient, Parallel-Group Study to Assess the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and ...
The purpose of the study is to assess the success of a single administration of Staccato alprazolam compared with placebo both in rapidly terminating a seizure episode within 90 seconds ...