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Last update: Tuesday July 08, 2025
Orphanet Journal of Rare Diseases
Chronic inflammatory demyelinating-like polyradiculoneuropathy (CIDP) is an autoimmune disease of the peripheral nervous system affecting myelin. CIDP is rare, with a reported prevalence...
Original:  Diagnosis of hereditary transthyretin amyloidosis in patients with suspected chronic...
Author(s): Péréon Yann et al.
 
Amyloid, March, 1–4.
We present the case of a 63-year-old woman patient with renal biopsy-proven ATTRv (hereditary transthyretin amyloidosis) and nephrotic syndrome presentation treated with patisiran after...
Original:  Complete Remission after Patisiran Treatment in a Patient with Nephrotic Syndrome...
Author(s): Vila et al.
 
International journal of cardiology. Heart & vasculature
The Val30Met (V30M) and Val122Ile (V122I) transthyretin (TTR) mutations often beget hereditary amyloid transthyretin amyloidosis (hATTR). Since symptoms are progressively debilitating...
Original:  Associations between pathophysiological traits and symptom development in retrospective...
Author(s): Kini, Sameer U. et al.

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Journal of the American College of Cardiology
Vutrisiran Improves Survival and Reduces Cardiovascular...  | Witteles, Ronald M. et al.
Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) have high mortality and morbidity. Vutrisiran, a subcutaneous...
Amyloid
Global patterns of amyloid typing: results of a survey...  | Hironobu Naiki et al.
Accurate tissue typing in amyloidosis is essential to provide appropriate therapy for individual patients. To get a real-life...
Journal of neurology
Elevated serum concentrations of GFAP in hereditary...  | Manco Carlo et al.
Hereditary transthyretin amyloidosis (ATTRv) is a rare disorder caused by pathogenic TTR gene variants. Glial fibrillary...
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This bibliographical database is provided by GNM Healthcare Consulting France SAS in full editorial independence. The information published within the framework of this service may discuss or suggest certain therapies that have not been validated by the competent national authorities.
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