Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis

(2019) Scott D. Solomon et al.   CIRCULATION

Burden of Hereditary Transthyretin Amyloidosis on Quality of Life

(2019) Aaron Yarlas et al.   MUSCLE & NERVE

Natural History, Quality of Life, and Outcome in Cardiac Transthyretin Amyloidosis

(2019) Thirusha Lane et al.   CIRCULATION

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

(2019) David Adams et al.   Nature Reviews Neurology

Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis)

(2018) Alejandra Gonzalez-Duarte   CLINICAL AUTONOMIC RESEARCH

Symptoms of Autonomic Dysfunction in Systemic Sclerosis Assessed by the COMPASS-31 Questionnaire

(2018) Brittany L. Adler et al.   JOURNAL OF RHEUMATOLOGY

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

Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial

(2017) Peter J. Dyck et al.   MUSCLE & NERVE

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

The Combined Effect of Neuropsychological and Neuropathological Deficits on Instrumental Activities of Daily Living in Older Adults: a Systematic Review

(2016) Eduard J. Overdorp et al.   NEUROPSYCHOLOGY REVIEW

Hereditary ATTR amyloidosis: a single-institution experience with 266 patients

(2015) Paul L. Swiecicki et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Evolving landscape in the management of transthyretin amyloidosis

(2015) Philip N. Hawkins et al.   ANNALS OF MEDICINE

Detection of undisclosed neuropathy and assessment of its impact on quality of life: a survey in 25,000 Romanian patients with diabetes

(2015) Andrei I. Veresiu et al.   JOURNAL OF DIABETES AND ITS COMPLICATIONS

Characterization of Pain in Familial Amyloid Polyneuropathy

(2015) Sophie Ng Wing Tin et al.   JOURNAL OF PAIN

The Val30Met familial amyloid polyneuropathy specific Rasch-built overall disability scale (FAP-RODS©)

(2015) Mariëlle H. J. Pruppers et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Rapid progression of familial amyloidotic polyneuropathy

(2015) David Adams et al.   NEUROLOGY

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

(2015) Ole B Suhr et al.   Orphanet Journal of Rare Diseases

Transthyretin Familial Amyloid Polyneuropathy Impact on Health-Related Quality of Life

(2015) M Inês et al.   VALUE IN HEALTH

Cardiac Findings and Events Observed in an Open-Label Clinical Trial of Tafamidis in Patients with non-Val30Met and non-Val122Ile Hereditary Transthyretin Amyloidosis

(2015) Thibaud Damy et al.   Journal of Cardiovascular Translational Research

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

(2015) John L. Berk et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

The Relative Impacts of Disease on Health Status and Capability Wellbeing: A Multi-Country Study

(2015) Paul Mark Mitchell et al.   PLoS One

Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy

(2014) Etta J. Vinik et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

THAOS: Gastrointestinal manifestations of transthyretin amyloidosis - common complications of a rare disease

(2014) Jonas Wixner et al.   Orphanet Journal of Rare Diseases

Cardiac amyloidosis: Updates in diagnosis and management

(2013) Dania Mohty et al.   Archives of Cardiovascular Diseases

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Guideline of transthyretin-related hereditary amyloidosis for clinicians

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

Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: The Transthyretin Amyloidosis Cardiac Study (TRACS)

(2012) Frederick L. Ruberg et al.   AMERICAN HEART JOURNAL

Transthyretin (TTR) Cardiac Amyloidosis

(2012) Frederick L. Ruberg et al.   CIRCULATION

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

Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective

(2012) C. Rapezzi et al.   EUROPEAN HEART JOURNAL

Cardiac transthyretin amyloidosis

(2012) Jason N Dungu et al.   HEART

COMPASS 31: A Refined and Abbreviated Composite Autonomic Symptom Score

(2012) David M. Sletten et al.   MAYO CLINIC PROCEEDINGS

Amyloid Neuropathies

(2012) Susan C. Shin et al.   MOUNT SINAI JOURNAL OF MEDICINE

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

(2012) T. Coelho et al.   NEUROLOGY

Recent advances in the treatment of familial amyloid polyneuropathy

(2012) David Adams   Therapeutic Advances in Neurological Disorders

Efficacy and Safety of Antioxidant Treatment With  -Lipoic Acid Over 4 Years in Diabetic Polyneuropathy: The NATHAN 1 trial

(2011) D. Ziegler et al.   DIABETES CARE

Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant

(2011) P. T. Sattianayagam et al.   EUROPEAN HEART JOURNAL

Rasch-built Overall Disability Scale (R-ODS) for immune-mediated peripheral neuropathies

(2011) S. I. van Nes et al.   NEUROLOGY