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Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Primary Purpose

FAP, Familial Amyloid Polyneuropathy, TTR

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Inotersen
Placebo
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for FAP focused on measuring FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

Eligibility Criteria

18 Years - 82 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Stage 1 and Stage 2 FAP participants with the following:

    1. NIS score within protocol criteria
    2. Documented transthyretin variant by genotyping
    3. Documented amyloid deposit by biopsy
  • Females of child-bearing potential must use appropriate contraception and be non-pregnant and non-lactating. Males engaging in relations of child-bearing potential are to use appropriate contraception

Exclusion Criteria:

  • Low Retinol level at screen
  • Karnofsky performance status ≤50
  • Poor Renal function
  • Known type 1 or type 2 diabetes mellitus
  • Other causes of sensorimotor or autonomic neuropathy (for example, autoimmune disease)
  • If previously treated with Vyndaqel®, will need to have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, will need to have discontinued treatment for 3 days prior to Study Day 1
  • Previous treatment with any oligonucleotide or siRNA within 12 months of screening
  • Prior liver transplant or anticipated liver transplant within 1 year of screening
  • New York Heart Association (NYHA) functional classification of ≥3
  • Acute Coronary Syndrome or major surgery within 3 months of screening
  • Known Primary or Leptomeningeal Amyloidosis
  • Anticipated survival less than 2 years
  • Any other conditions in the opinion of the investigator which interfere with the participant participating in or completing the study

Sites / Locations

  • University of California, Irvine
  • Indiana University School of Medicine
  • Johns Hopkins University Bayview Medical Center
  • Boston University School of Medicine - Amyloid Treatment & Research Program
  • Mayo Clinic
  • Mount Sinai Medical Center
  • Columbia University Medical Center - The Neurological Institute
  • Oregon Health & Science University
  • Penn Presbyterian Medical Center
  • FLENI
  • Federal University of Rio de Janeiro - University Hospital
  • AACD
  • UNIFESP
  • CHU Henri Mondor - Department of Neurology
  • CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network
  • UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin
  • Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"
  • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo
  • Auckland City Hospital
  • CHLN - Hospital de Santa Maria
  • CHP-HGSA, Unidade Clinica de Paramiloidose
  • Hospital Universitari Vall D' Hebron
  • Hospital Clínic
  • University College London - National Amyloidosis Centre

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Inotersen

Placebo

Arm Description

300 mg inotersen administered subcutaneously (SC) 3 times on alternate days in the first week and then once-weekly for 64 weeks

Placebo administered SC 3 times on alternate days in the first week and then once-weekly for 64 weeks

Outcomes

Primary Outcome Measures

Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66
The mNIS+7 composite score is a measure of neurologic impairment that evaluates muscle weakness, sensation, reflexes, nerve conduction, and autonomic function. The mNIS+7 Composite Score has a range of -22.32 to 346.32 and a higher mNIS+7 composite score indicates lower function.
Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66
The Norfolk QoL-DN score is a measure of physical function/large fiber neuropathy, symptoms, activities of daily living, small fiber neuropathy, and autonomic neuropathy. The Norfolk QoL-DN total score has a range of -4 to 136, and a higher Norfolk QoL-DN score indicates poorer QoL.

Secondary Outcome Measures

Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66
The Norfolk QoL-DN symptoms score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN symptoms domain score has a range of 0-32, and a higher Norfolk QoL-DN score indicates poorer QoL.
Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66
The Norfolk QoL-DN physical functioning/large fiber neuropathy domain score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN physical function/large fiber neuropathy domain score has a range of -4 to 56, and a higher Norfolk QoL-DN domain score indicates poorer QoL.
Change From Baseline In Modified Body Mass Index (mBMI) at Week 65
The mBMI is the BMI multiplied by the serum albumin g/L
Change From Baseline In Body Mass Index (BMI) at Week 65
Change From Baseline in Neuropathy Impairment Score (NIS) at Week 66
The NIS score is a measure of neurologic impairment. The NIS Score has a range of 0 to 244 and a higher NIS score indicates lower function.
Change From Baseline in Modified +7 at Week 66
The Modified +7 score is a version of the NIS score that is a measure of neurologic impairment. The Modified +7 Score has a range of -22.32 to 102.32 and a higher NIS score indicates lower function.
Change From Baseline in NIS+7 at Week 66
The NIS+7 score is a version of the NIS score that is a measure of neurologic impairment. The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS score indicates lower function.
Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) at Week 65 in the CM-ECHO Set
GLS by ECHO is a measure of cardiac systolic function
Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram ECHO at Week 65 in the ECHO Subgroup
GLS by ECHO is a measure of cardiac systolic function
Change From Baseline in Transthyretin (TTR) Level at Week 65
Change From Baseline in Retinol Binding Protein 4 (RBP4) Level at Week 65
Maximum Measured Plasma Concentration (Cmax) Of Inotersen At Week 65
Time To The Maximum Plasma Concentration (Tmax) Of Inotersen At Week 65
Area Under The Plasma Concentration-time Curve From 0 To 24 Hours (AUC[0-24hr]) Of Inotersen At Week 65
Area Under The Plasma Concentration-time Curve From 0 To 168 Hours (AUC[0-168hr]) Of Inotersen At Week 65
Plasma Clearance From 0 To 24 Hours (CL[0-24hr]/F) Of Inotersen At Week 65
Inotersen Plasma Clearance At Steady State (CLss/F) At Week 65

Full Information

First Posted
November 27, 2012
Last Updated
July 8, 2019
Sponsor
Ionis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01737398
Brief Title
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
Official Title
A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
March 15, 2013 (Actual)
Primary Completion Date
March 3, 2017 (Actual)
Study Completion Date
November 7, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).
Detailed Description
FAP is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP. Inotersen (also known as ISIS 420915) is an antisense drug that was designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein would result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression. The purpose of this study is to determine if inotersen can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP participants. Participants will receive either inotersen or placebo for 65 weeks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Keywords
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
173 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Inotersen
Arm Type
Active Comparator
Arm Description
300 mg inotersen administered subcutaneously (SC) 3 times on alternate days in the first week and then once-weekly for 64 weeks
Arm Title
Placebo
Arm Type
Active Comparator
Arm Description
Placebo administered SC 3 times on alternate days in the first week and then once-weekly for 64 weeks
Intervention Type
Drug
Intervention Name(s)
Inotersen
Other Intervention Name(s)
TEGSEDI, IONIS-TTR Rx, ISIS 420915
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66
Description
The mNIS+7 composite score is a measure of neurologic impairment that evaluates muscle weakness, sensation, reflexes, nerve conduction, and autonomic function. The mNIS+7 Composite Score has a range of -22.32 to 346.32 and a higher mNIS+7 composite score indicates lower function.
Time Frame
Baseline and Week 66
Title
Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66
Description
The Norfolk QoL-DN score is a measure of physical function/large fiber neuropathy, symptoms, activities of daily living, small fiber neuropathy, and autonomic neuropathy. The Norfolk QoL-DN total score has a range of -4 to 136, and a higher Norfolk QoL-DN score indicates poorer QoL.
Time Frame
Baseline and Week 66
Secondary Outcome Measure Information:
Title
Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66
Description
The Norfolk QoL-DN symptoms score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN symptoms domain score has a range of 0-32, and a higher Norfolk QoL-DN score indicates poorer QoL.
Time Frame
Baseline and Week 66
Title
Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66
Description
The Norfolk QoL-DN physical functioning/large fiber neuropathy domain score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN physical function/large fiber neuropathy domain score has a range of -4 to 56, and a higher Norfolk QoL-DN domain score indicates poorer QoL.
Time Frame
Baseline and Week 66
Title
Change From Baseline In Modified Body Mass Index (mBMI) at Week 65
Description
The mBMI is the BMI multiplied by the serum albumin g/L
Time Frame
Baseline and Week 65
Title
Change From Baseline In Body Mass Index (BMI) at Week 65
Time Frame
Baseline and Week 65
Title
Change From Baseline in Neuropathy Impairment Score (NIS) at Week 66
Description
The NIS score is a measure of neurologic impairment. The NIS Score has a range of 0 to 244 and a higher NIS score indicates lower function.
Time Frame
Baseline and Week 66
Title
Change From Baseline in Modified +7 at Week 66
Description
The Modified +7 score is a version of the NIS score that is a measure of neurologic impairment. The Modified +7 Score has a range of -22.32 to 102.32 and a higher NIS score indicates lower function.
Time Frame
Baseline and Week 66
Title
Change From Baseline in NIS+7 at Week 66
Description
The NIS+7 score is a version of the NIS score that is a measure of neurologic impairment. The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS score indicates lower function.
Time Frame
Baseline and Week 66
Title
Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) at Week 65 in the CM-ECHO Set
Description
GLS by ECHO is a measure of cardiac systolic function
Time Frame
Baseline and Week 65
Title
Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram ECHO at Week 65 in the ECHO Subgroup
Description
GLS by ECHO is a measure of cardiac systolic function
Time Frame
Baseline and Week 65
Title
Change From Baseline in Transthyretin (TTR) Level at Week 65
Time Frame
Baseline and Week 65
Title
Change From Baseline in Retinol Binding Protein 4 (RBP4) Level at Week 65
Time Frame
Baseline and Week 65
Title
Maximum Measured Plasma Concentration (Cmax) Of Inotersen At Week 65
Time Frame
Week 65
Title
Time To The Maximum Plasma Concentration (Tmax) Of Inotersen At Week 65
Time Frame
Week 65
Title
Area Under The Plasma Concentration-time Curve From 0 To 24 Hours (AUC[0-24hr]) Of Inotersen At Week 65
Time Frame
Week 65
Title
Area Under The Plasma Concentration-time Curve From 0 To 168 Hours (AUC[0-168hr]) Of Inotersen At Week 65
Time Frame
Week 65
Title
Plasma Clearance From 0 To 24 Hours (CL[0-24hr]/F) Of Inotersen At Week 65
Time Frame
Week 65
Title
Inotersen Plasma Clearance At Steady State (CLss/F) At Week 65
Time Frame
Week 65

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
82 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Stage 1 and Stage 2 FAP participants with the following: NIS score within protocol criteria Documented transthyretin variant by genotyping Documented amyloid deposit by biopsy Females of child-bearing potential must use appropriate contraception and be non-pregnant and non-lactating. Males engaging in relations of child-bearing potential are to use appropriate contraception Exclusion Criteria: Low Retinol level at screen Karnofsky performance status ≤50 Poor Renal function Known type 1 or type 2 diabetes mellitus Other causes of sensorimotor or autonomic neuropathy (for example, autoimmune disease) If previously treated with Vyndaqel®, will need to have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, will need to have discontinued treatment for 3 days prior to Study Day 1 Previous treatment with any oligonucleotide or siRNA within 12 months of screening Prior liver transplant or anticipated liver transplant within 1 year of screening New York Heart Association (NYHA) functional classification of ≥3 Acute Coronary Syndrome or major surgery within 3 months of screening Known Primary or Leptomeningeal Amyloidosis Anticipated survival less than 2 years Any other conditions in the opinion of the investigator which interfere with the participant participating in or completing the study
Facility Information:
Facility Name
University of California, Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Indiana University School of Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Johns Hopkins University Bayview Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Boston University School of Medicine - Amyloid Treatment & Research Program
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Mount Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Columbia University Medical Center - The Neurological Institute
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn Presbyterian Medical Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
FLENI
City
Buenos Aires
Country
Argentina
Facility Name
Federal University of Rio de Janeiro - University Hospital
City
Rio de Janeiro
ZIP/Postal Code
CEP 21941913
Country
Brazil
Facility Name
AACD
City
Sao Paulo
Country
Brazil
Facility Name
UNIFESP
City
Sao Paulo
Country
Brazil
Facility Name
CHU Henri Mondor - Department of Neurology
City
Creteil
ZIP/Postal Code
94000
Country
France
Facility Name
CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network
City
Le Kremlin Bicetre
ZIP/Postal Code
94275
Country
France
Facility Name
UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin
City
Munster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"
City
Messina
State/Province
Sicily
ZIP/Postal Code
98124
Country
Italy
Facility Name
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Auckland City Hospital
City
Auckland
Country
New Zealand
Facility Name
CHLN - Hospital de Santa Maria
City
Lisbon
ZIP/Postal Code
1649-035
Country
Portugal
Facility Name
CHP-HGSA, Unidade Clinica de Paramiloidose
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Facility Name
Hospital Universitari Vall D' Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Clínic
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
University College London - National Amyloidosis Centre
City
London
ZIP/Postal Code
NW3 2PF
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
29972757
Citation
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Plante-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceicao I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.
Results Reference
result
PubMed Identifier
35799473
Citation
Karam C, Brown D, Yang M, Done N, Zhu JJ, Greatsinger A, Bozas A, Vera-Llonch M, Signorovitch J. Long-term treatment effects of inotersen on health-related quality of life in patients with hATTR amyloidosis with polyneuropathy: Analysis of the open-label extension of the NEURO-TTR trial. Muscle Nerve. 2022 Oct;66(4):438-446. doi: 10.1002/mus.27675. Epub 2022 Aug 4.
Results Reference
derived
PubMed Identifier
35766224
Citation
Karam C, Brown D, Yang M, Done N, Dieye I, Bozas A, Vera Llonch M, Signorovitch J. Factors associated with increased health-related quality-of-life benefits in hereditary transthyretin amyloidosis polyneuropathy patients treated with inotersen. Muscle Nerve. 2022 Sep;66(3):319-328. doi: 10.1002/mus.27668. Epub 2022 Jul 15.
Results Reference
derived
PubMed Identifier
34355354
Citation
Yarlas A, Lovley A, McCausland K, Brown D, Vera-Llonch M, Conceicao I, Karam C, Khella S, Obici L, Waddington-Cruz M. Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR. Neurol Ther. 2021 Dec;10(2):865-886. doi: 10.1007/s40120-021-00268-x. Epub 2021 Aug 5.
Results Reference
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PubMed Identifier
34125267
Citation
Yarlas A, Lovley A, Brown D, Kosinski M, Vera-Llonch M. Responder analysis for neuropathic impairment and quality-of-life assessment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTR study. J Neurol. 2022 Jan;269(1):323-335. doi: 10.1007/s00415-021-10635-1. Epub 2021 Jun 14.
Results Reference
derived
PubMed Identifier
32833564
Citation
Yu RZ, Wang Y, Norris DA, Kim TW, Narayanan P, Geary RS, Monia BP, Henry SP. Immunogenicity Assessment of Inotersen, a 2'-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety. Nucleic Acid Ther. 2020 Oct;30(5):265-275. doi: 10.1089/nat.2020.0867. Epub 2020 Aug 19.
Results Reference
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PubMed Identifier
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Citation
Dyck PJB, Kincaid JC, Wiesman JF, Polydefkis M, Litchy WJ, Mauermann ML, Ackermann EJ, Guthrie S, Pollock M, Jung SW, Baker BF, Dyck PJ. mNIS+7 and lower limb function in inotersen treatment of hereditary transthyretin-mediated amyloidosis. Muscle Nerve. 2020 Oct;62(4):502-508. doi: 10.1002/mus.27022. Epub 2020 Aug 13.
Results Reference
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PubMed Identifier
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Citation
Dyck PJB, Coelho T, Waddington Cruz M, Brannagan TH 3rd, Khella S, Karam C, Berk JL, Polydefkis MJ, Kincaid JC, Wiesman JF, Litchy WJ, Mauermann ML, Ackermann EJ, Baker BF, Jung SW, Guthrie S, Pollock M, Dyck PJ. Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis. Muscle Nerve. 2020 Oct;62(4):509-515. doi: 10.1002/mus.27023. Epub 2020 Aug 7.
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PubMed Identifier
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Citation
Coelho T, Yarlas A, Waddington-Cruz M, White MK, Sikora Kessler A, Lovley A, Pollock M, Guthrie S, Ackermann EJ, Hughes SG, Karam C, Khella S, Gertz M, Merlini G, Obici L, Schmidt HH, Polydefkis M, Dyck PJB, Brannagan Iii TH, Conceicao I, Benson MD, Berk JL. Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis. J Neurol. 2020 Apr;267(4):1070-1079. doi: 10.1007/s00415-019-09671-9. Epub 2019 Dec 18.
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Citation
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Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

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