Nykobing Sj�lland, Denmark

A Phase 3 Study of NTLA-2001 in ATTRv-PN

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 50 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.

AZD0901 Compared With Investigator's Choice of Therapy in Participants With Second- or Later-line Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma Expressing Claudin18.2

This is a Phase III, multi-center, open-label, sponsor-blinded, randomized, global study to assess the efficacy and safety of AZD0901 compared to Investigator's choice of therapy as the 2L+ treatment for participants with advanced or metastatic gastric or GEJ adenocarcinoma expressing CLDN18.2, and the clinical performance of the investigational IVD. As part of this combined approach, the efficacy analyses from this study will also provide the basis to evaluate the clinical performance of Ventana CLDN18.2 assay as an IVD device for the identification of patients with advanced or metastatic gastric or GEJ adenocarcinoma expressing CLDN18.2 who may benefit from AZD0901.

Study to Evaluate the Efficacy and Safety of K-877-ER and CSG452 in Participants With NASH With Liver Fibrosis

A Study Comparing Talquetamab in Combination With Daratumumab or in Combination With Daratumumab and Pomalidomide Versus Daratumumab in Combination With Pomalidomide and Dexamethasone in Participants With Multiple Myeloma That Returns After Treatment or is Resistant to Treatment

Feasibility Study of Intermediate Doses of ARA-C With Autologous SCT as Consolidation of Low/Intermediate-risk AML

1. Compare overall survival and disease-free survival of patients with acute myeloid leukemia classified according the European LeukemiaNet treated in participating South American hospitals with the results reported in developed countries. 2. Compare overall survival and disease-free survival of patients with AML low or intermediate risk treated with two cycles of cytarabine in intermediate dose versus one cytarabine cycle at the same dose followed by autologous SCT as consolidation. The risk will be established according to the classification of the European LeukemiaNet. 3. Create a network of institutions in developing countries that will perform AML diagnosis, risk classification, treatment, supportive care and follow-up evaluation according to a common protocol and will register data using common clinical research forms (CRFs) in a single database and available on the internet 4. Using National Reference Laboratories, provide cytogenetic and molecular methods for all institutions participating in the network, thus allowing rapid diagnosis and risk stratification of AML cases according to the European LeukemiaNet structure; 5. Develop a method of assessing minimal residual disease based on flow cytometry adapted to local resources and capable of guiding therapeutic decisions; 6. Determine the time interval between: a) diagnosis and risk group determination; b) the first cycle of consolidation chemotherapy and autologous hematopoietic stem cells infusion; 7. Determine the frequency and etiologic agent of infections associated with treatment, the number and average duration of hospitalization due to episodes of neutropenia; 8. Create a bank of samples of bone marrow from AML patients at different times of treatment; 9. Determine the disease-free survival and the cumulative incidence rate of relapse and non-relapse mortality and compare them between chemotherapy alone and chemotherapy plus autologous SCT cohorts.

Study of TSR-042, an Anti-programmed Cell Death-1 Receptor (PD-1) Monoclonal Antibody, in Participants With Advanced Solid Tumors

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

A Study of Teclistamab in Combination With Daratumumab and Lenalidomide (Tec-DR) and Talquetamab in Combination With Daratumumab and Lenalidomide (Tal-DR) in Participants With Newly Diagnosed Multiple Myeloma

Pompe Disease Registry Protocol

Study Design Time Perspective: Retrospective and Prospective

Open Label, Long-term Study Evaluating Safety and Efficacy of Subcutaneous Amlitelimab in Participants Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis