Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study - Wikidata - awgldk - TriplyDB

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

http://www.wikidata.org/entity/Q36025267

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Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells.The delivery of therapeutic oligonucleotides Systemic delivery of factor IX messenger RNA for protein replacement therapy Cardiac amyloidosis: An update on pathophysiology, diagnosis, and treatment.Advances in the delivery of RNA therapeutics: from concept to clinical reality.TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies.Novel pharmacotherapies for cardiac amyloidosis.Causally treatable, hereditary neuropathies in Fabry's disease, transthyretin-related familial amyloidosis, and Pompe's disease.Current and Future Treatment Approaches in Transthyretin Familial Amyloid Polyneuropathy.From the RNA world to the clinic.One mutation, two distinct disease variants: unravelling the impact of transthyretin amyloid fibril composition.Oligonucleotide Therapy for Obstructive and Restrictive Respiratory Diseases.The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market.Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.Genetic and clinical characteristics of hereditary transthyretin amyloidosis in endemic and non-endemic areas: experience from a single-referral center in Japan.RNA Therapeutics in Oncology: Advances, Challenges, and Future Directions.The impact of selection bias in randomized multi-arm parallel group clinical trials.Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Management of gastrointestinal complications in hereditary transthyretin amyloidosis: a single-center experience over 40 years.Cognitive impairment in liver transplanted patients with transthyretin-related hereditary amyloid polyneuropathy.Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.Development of Controlled Drug Delivery Systems for Bone Fracture-Targeted Therapeutic Delivery: A Review.Small Fiber Neuropathy: Disease Classification Beyond Pain and Burning.Wild-type transthyretin cardiac amyloidosis (ATTRwt-CA), previously known as senile cardiac amyloidosis: clinical presentation, diagnosis, management and emerging therapies.RNA Therapeutics in Cardiovascular Precision Medicine Evaluation of Amino-Functional Polyester Dendrimers Based on Bis-MPA as Nonviral Vectors for siRNA Delivery

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Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

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Brian R Bettencourt

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Teresa Coelho

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P2860

Fibril in senile systemic amyloidosis is derived from normal transthyretin

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial

Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial

Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

Leukocytosis: basics of clinical assessment.

Outcome in systemic AL amyloidosis in relation to changes in concentration of circulating free immunoglobulin light chains following chemotherapy.

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial.

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