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Familial Amyloid Neuropathies (Familial Amyloid Polyneuropathy)

Inherited disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. The different clinical types based on symptoms correspond to the presence of a variety of mutations in several different proteins including transthyretin (PREALBUMIN); APOLIPOPROTEIN A-I; and GELSOLIN.
Also Known As:
Familial Amyloid Polyneuropathy; Amyloid Neuropathies, Familial; Amyloid Neuropathy Type 1; Amyloid Polyneuropathy, British Type; Amyloid Polyneuropathy, Iowa Type; Amyloid Polyneuropathy, Swiss Type; Appalachian Type Familial Amyloid Polyneuropathy; British Type Amyloid Polyneuropathy; Cerebral Amyloid Angiopathy, British Type; Familial Amyloid Neuropathy, Andrade Type; Familial Amyloid Neuropathy, Finnish Type; Familial Amyloid Neuropathy, Portuguese Type; Familial Amyloid Polyneuropathy, Appalachian Type; Familial Amyloid Polyneuropathy, Jewish Type; Familial Amyloid Polyneuropathy, Type I; Familial Amyloid Polyneuropathy, Type II; Familial Amyloid Polyneuropathy, Type III; Familial Amyloid Polyneuropathy, Type IV; Familial Amyloid Polyneuropathy, Type V; Familial Amyloid Polyneuropathy, Type VI; Familial Portuguese Polyneuritic Amyloidosis; Finnish Type Familial Amyloid Neuropathy; Hereditary Neuropathic Amyloidosis; Iowa Type Amyloid Polyneuropathy; Jewish Type Familial Amyloid Polyneuropathy; Neuropathic Amyloid Syndrome; Polyneuritic Amyloidosis, Portuguese; Portuguese Polyneuritic Amyloidosis; Portuguese Type Familial Amyloid Neuropathy; Swiss Type Amyloid Polyneuropathy; Type I Familial Amyloid Polyneuropathy; Type II Familial Amyloid Polyneuropathy; Type III Familial Amyloid Polyneuropathy; Type IV Familial Amyloid Polyneuropathy; Type V Familial Amyloid Polyneuropathy; Type VI Familial Amyloid Polyneuropathy; Wohlwill-Andrade Syndrome; Wohlwill-Corino Andrade Syndrome; Amyloid Neuropathy, Familial; Amyloid Polyneuropathies, Familial; Amyloid Polyneuropathy, Familial; Amyloid Syndrome, Neuropathic; Amyloid Syndromes, Neuropathic; Amyloidoses, Hereditary Neuropathic; Amyloidoses, Portuguese Polyneuritic; Amyloidosis, Hereditary Neuropathic; Amyloidosis, Portuguese Polyneuritic; Familial Amyloid Neuropathy; Hereditary Neuropathic Amyloidoses; Neuropathic Amyloid Syndromes; Neuropathic Amyloidoses, Hereditary; Neuropathic Amyloidosis, Hereditary; Neuropathies, Familial Amyloid; Neuropathy, Familial Amyloid; Polyneuritic Amyloidoses, Portuguese; Polyneuropathies, Familial Amyloid; Polyneuropathy, Familial Amyloid; Portuguese Polyneuritic Amyloidoses; Wohlwill Andrade Syndrome; Wohlwill Corino Andrade Syndrome; Familial Amyloid Polyneuropathies
Networked: 656 relevant articles (17 outcomes, 45 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Polyneuropathies (Polyneuropathy)
2. Amyloidosis
3. Cardiomyopathies (Cardiomyopathy)
4. Orthostatic Hypotension (Postural Hypotension)
5. Multiple System Atrophy

Experts

1. Ando, Yukio: 34 articles (01/2020 - 11/2002)
2. Coelho, Teresa: 32 articles (01/2021 - 03/2004)
3. Sekijima, Yoshiki: 21 articles (02/2021 - 03/2003)
4. Ueda, Mitsuharu: 21 articles (01/2020 - 01/2005)
5. Ikeda, Shu-ichi: 21 articles (12/2013 - 11/2002)
6. Koike, Haruki: 18 articles (05/2019 - 11/2002)
7. Adams, David: 18 articles (01/2019 - 07/2004)
8. Yamashita, Taro: 17 articles (07/2020 - 01/2005)
9. Kelly, Jeffery W: 16 articles (01/2021 - 04/2002)
10. Conceição, Isabel: 16 articles (07/2020 - 06/2002)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Familial Amyloid Neuropathies:
1. Prealbumin (Transthyretin)IBA
2. tafamidisIBA
3. Proteins (Proteins, Gene)FDA Link
4. Amyloid (Amyloid Fibrils)IBA
5. Diflunisal (Dolobid)FDA Link
6. Dopamine beta-HydroxylaseIBA
7. 3-Iodobenzylguanidine (Iobenguane)IBA
8. RNA (Ribonucleic Acid)IBA
12/01/2016 - "Finally, the era of genetic therapy appears to have come to fruition with the interfering RNA trial for familial amyloid neuropathies."
04/01/2013 - "We report a 31-year-old woman with familial amyloid polyneuropathy who received a living-donor liver transplant from her husband who was hepatitis C virus antibody-positive but HCV-RNA negative and ABO incompatible. "
04/01/2013 - "Liver transplant from an ABO-incompatible and hepatitis C antibody-positive but an HCV-RNA negative living donor in a familial amyloid polyneuropathy patient."
01/01/2022 - " 2'-MOE: 2'-O-(2-methoxyethyl); 2'-O-Me: 2'-O-methyl; 2'-F: 2'-fluoro; AD: Alzheimer's disease; ALS: Amyotrophic lateral sclerosis; ALSFRS-R: Revised Amyotrophic Lateral Sclerosis Functional Rating Scale; ARC: Antibody siRNA Conjugate; AS: Angelman Syndrome; ASGRP: Asialoglycoprotein receptor; ASO: Antisense oligonucleotide; AxD: Alexander Disease; BBB: Blood brain barrier; Bp: Basepair; CNM: Centronuclear myopathies; CNS: Central Nervous System; CPP: Cell-penetrating Peptide; CSF: Cerebrospinal fluid; DMD: Duchenne muscular dystrophy; DNA: Deoxyribonucleic acid; FAP: Familial amyloid polyneuropathy; FALS: Familial amyotrophic lateral sclerosis; FDA: The United States Food and Drug Administration; GalNAc: N-acetylgalactosamine; GoF: Gain of function; hATTR: Hereditary transthyretin amyloidosis; HD: Huntington's disease; HRQOL: health-related quality of life; ICV: Intracerebroventricular; IT: Intrathecal; LNA: Locked nucleic acid; LoF: Loss of function; mRNA: Messenger RNA; MS: Multiple Sclerosis; MSA: Multiple System Atrophy; NBE: New Biological Entity; NCE: New Chemical Entity; NHP: Nonhuman primate; nt: Nucleotide; PD: Parkinson's disease; PNP: Polyneuropathy; PNS: Peripheral nervous system; PS: Phosphorothioate; RISC: RNA-Induced Silencing Complex; RNA: Ribonucleic acid; RNAi: RNA interference; s.c.: Subcutaneous; siRNA: Small interfering RNA; SMA: Spinal muscular atrophy; SMN: Survival motor neuron; TTR: Transthyretin."
9. Peptides (Polypeptides)IBA
10. OligonucleotidesIBA

Therapies and Procedures

1. Liver Transplantation
2. Therapeutics
3. Transplantation
4. Spinal Cord Stimulation
5. Sympathectomy (Sympathectomies)