Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
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Therapeutic Oligonucleotides Targeting Liver Disease: TTR AmyloidosisEvaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells.The delivery of therapeutic oligonucleotidesSystemic delivery of factor IX messenger RNA for protein replacement therapyCardiac 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 MedicineEvaluation of Amino-Functional Polyester Dendrimers Based on Bis-MPA as Nonviral Vectors for siRNA Delivery
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P2860
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@ast
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@en
type
label
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@ast
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@en
prefLabel
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@ast
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@en
P2093
P2860
P50
P1476
Efficacy and safety of patisir ...... y: a phase II multi-dose study
@en
P2093
Akshay Vaishnaw
Brian R Bettencourt
Jared Gollob
Jean Pouget
Josep M Campistol
Juan Buades
Márcia Waddington-Cruz
Teresa Coelho
P2860
P2888
P356
10.1186/S13023-015-0326-6
P577
2015-09-04T00:00:00Z
P5875
P6179
1043613539