Clinical Trials and Research

Change point analysis of the total PYP orders showed a significant difference in the number of PYP orders

Increasing clinicians’ suspicion of ATTR amyloidosis using a retrospective algorithm

AstraZeneca advances scientific leadership in haematology at ASH 2023

Calquence six-year follow-up data reinforce long-term benefit in chronic lymphocytic leukaemia, and data across multiple haematology assets showcase breadth of…

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Published: Oct 09, 2023 - FDA Cites Insufficient Evidence of Clinical Meaningfulness - - No Clinical Safety, Study Conduct, Drug Quality or Manufacturing…

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting

- Cardiovascular and Renal Drugs Advisory Committee Voted 9:3 That the Benefits of Patisiran Outweigh its Risks for the Treatment of the Cardiomyopathy of ATTR…

Nexcella, Inc. Announces Additional Positive NXC-201 Clinical Data Demonstrating 100% Complete Responses in Relapsed/Refractory AL Amyloidosis Patients, Duration of Response Not Yet Reached

Nexcella, Inc., a biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, today announced…

Prothena Presents Data Demonstrating Consistent Survival Benefit Observed with Birtamimab in Mayo Stage IV AL Amyloidosis Patients in Phase 3 VITAL Study at ASH 2022

Eplontersen Met Co-Primary and Secondary Endpoints in Interim Analysis of the NEURO-TTRansform Phase III Trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN)

Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed…

Positive New Data From Phase 1/2 Trial of AT-01 (Iodine I-124 Evuzamitide) Presented at 2022 Society of Nuclear Medicine & Molecular Imaging Annual Meeting

Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced…

Alnylam Announces FDA Approval of AMVUTTRA™ (Vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA(tm) (vutrisiran…

Alnylam Presents New 18-Month Results From Exploratory Cardiac Endpoints in HELIOS-A Phase 3 Study of Investigational Vutrisiran

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced new positive results from an 18-month analysis of exploratory cardiac…

BridgeBio Pharma Presents Updated Results From Phase 2 Open-Label Extension Study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced updated data from its ongoing Phase 2…

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