Health-related quality of life in hereditary transthyretin amyloidosis polyneuropathy: a prospective, observational study

Burden of Hereditary Transthyretin Amyloidosis on Quality of Life

(2019) Aaron Yarlas et al.   MUSCLE & NERVE

PCV123 DETERMINANTS OF POOR QUALITY OF LIFE AND HIGH HEALTH CARE RESOURCE CONSUMPTION IN HEART FAILURE PATIENTS IN PORTUGAL: AN ANALYSIS OF THE PRIME STUDY

(2019) M. Afonso-Silva et al.   VALUE IN HEALTH

Estimating the global prevalence of transthyretin familial amyloid polyneuropathy

(2018) Hartmut H. Schmidt et al.   MUSCLE & NERVE

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers

(2018) Daniel A. Ollendorf et al.   VALUE IN HEALTH

The Problem of Rarity: Estimation of Prevalence in Rare Disease

(2018) Stéphane Auvin et al.   VALUE IN HEALTH

Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group

(2018) Sandra Nestler-Parr et al.   VALUE IN HEALTH

Epidemiology of Transthyretin Familial Amyloid Polyneuropathy in Portugal: A Nationwide Study

(2018) Mónica Inês et al.   NEUROEPIDEMIOLOGY

Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy

(2018) Teresa Coelho et al.   NEUROLOGY

Health-related quality of life in adults with type 2 diabetes mellitus starting with new glucose lowering drugs: An inception cohort study

(2018) Carla Torre et al.   Primary Care Diabetes

Assessing Health-Related Quality Of Life In Patients With Psoriasis Compared With The Portuguese Population

(2017) LN Ferreira et al.   VALUE IN HEALTH

Measurement and control of bias in patient reported outcomes using multidimensional item response theory

(2016) N. Maritza Dowling et al.   BMC Medical Research Methodology

Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe

(2016) Yesim Parman et al.   CURRENT OPINION IN NEUROLOGY

“Red-flag” symptom clusters in transthyretin familial amyloid polyneuropathy

(2016) Isabel Conceição et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France

(2015) Louise-Laure Mariani et al.   ANNALS OF NEUROLOGY

FAP Neuropathy and Emerging Treatments

(2014) David Adams et al.   Current Neurology and Neuroscience Reports

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

(2013) Teresa Coelho et al.   JOURNAL OF NEUROLOGY

Guideline of transthyretin-related hereditary amyloidosis for clinicians

(2013) Yukio Ando et al.   Orphanet Journal of Rare Diseases

The valuation of the EQ-5D in Portugal

(2013) Lara N. Ferreira et al.   QUALITY OF LIFE RESEARCH

EQ-5D Portuguese population norms

(2013) Lara Noronha Ferreira et al.   QUALITY OF LIFE RESEARCH

The Transthyretin Amyloidosis Outcomes Survey (THAOS) registry: design and methodology

(2012) Violaine Planté-Bordeneuve et al.   CURRENT MEDICAL RESEARCH AND OPINION

THAOS – The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis

(2012) Teresa Coelho et al.   CURRENT MEDICAL RESEARCH AND OPINION

Liver transplantation and transthyretin amyloidosis

(2012) Merrill D. Benson   MUSCLE & NERVE

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

(2012) T. Coelho et al.   NEUROLOGY

Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)

(2010) Gösta Holmgren et al.   CLINICAL GENETICS

Using Instrument-Defined Health State Transitions to Estimate Minimally Important Differences for Four Preference-Based Health-Related Quality of Life Instruments

(2010) Nan Luo et al.   MEDICAL CARE

Psychosocial impact of presymptomatic genetic testing for transthyretin amyloidotic polyneuropathy

(2008) Anita Graceffa et al.   NEUROMUSCULAR DISORDERS