Hereditary Transthyretin-mediated Amyloidosis

• CLINICAL_TRIALS research · 2026-04-24
  Clinical Trial Update: Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

  Clinical trial update indicates that Alnylam's patisiran-LNP pregnancy surveillance program is currently recruiting participants for heredit…

• CLINICAL_TRIALS research · 2026-04-02
  Clinical Trial Update: Phenotypic Manifestations of Hereditary ATTR Amyloidosis

  A clinical trial to characterize the phenotypic manifestations of hereditary transthyretin-mediated amyloidosis; it is currently recruiting …

• CLINICAL_TRIALS research · 2025-12-24
  Clinical Trial Update: Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

  An observational study is evaluating neurofilament light chain as a biomarker in asymptomatic carriers of transthyretin variants and in pati…

• CLINICAL_TRIALS research · 2025-08-14
  Clinical Trial Update: Prospective Evaluation of NfL as a Biomarker in ATTRv

  This clinical trial update reports a prospective evaluation of neurofilament light chain (NfL) as a biomarker in hereditary transthyretin-me…

• CLINICAL_TRIALS research · 2025-04-06
  Clinical Trial Update: Polyneuropathy, Impairments and Physical Activity - The PolyImPAct Study

  A clinical trial update for the PolyImPAct study investigating polyneuropathy, impairments, and physical activity, currently recruiting and …

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• CLINICAL_TRIALS research · 2024-05-20
  Clinical Trial Update: Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

  Expanded access protocol for patisiran in hereditary transthyretin-mediated amyloidosis has been approved for marketing, enabling broader pa…