30 closest matches · ranked by relevance
Transthyretin Familial Amyloid Polyneuropathy
Primary: Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay — 10 Participants
Hereditary Transthyretin Amyloidosis (ATTRv) · Polyneuropathy
Primary: Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv — 1.6 Change in score*months
Transthyretin Gene Mutations · Transthyretin Amyloidosis
Primary: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 621; 175; 66; 4 Participants
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) · Transthyretin Cardiomyopathy (TTR-CM) · Familial Amyloid Cardiomyopathy
Primary: Demographical Characteristics of Participants — 20; 11; 39; 19 participants
Familial Amyloid Polyneuropathy
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of participants
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Primary: Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 — 2.3 Units on a scale
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Primary: Percentage of Participants With Cardiac Fixation at the Radionuclide Bone Scintigraphy and/or Single Photon Emission Computed Tomography (SPECT): FAS1 — 32.0 Percentage…
Amyloidosis, Familial · Transthyretin Amyloidosis
Primary: Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) — 91.0 percent reduction
ATTR-CM · TTR-CM
Primary: Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 12 — 0; 16.1; 9.7; 51.6 percentage of participants
Amyloidosis; Heart (Manifestation) · Senile Cardiac Amyloidosis
Primary: Changes in Strain Echocardiography — 9.1 percent LV shortening
TTR-mediated Amyloidosis
Primary: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation — 1; 3; 2; 7 Participants
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Primary: Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66 — 0.2964; 25.0557 scores on a scale — p=0.00000001
Familial Amyloid Polyneuropathy · ATTR-PN
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 — 62.2; 68.8 percentage of participants
Amyloidosis · Amyloid Cardiomyopathy · Transthyretin Amyloidosis
Primary: A Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT…
Transthyretin-associated Amyloidosis With Polyneuropathy
Primary: Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Week 6 — 94.7 percentage of participants
Cardiomyopathy
Primary: Percentage of Participants With Stabilized Transthyretin (TTR Tetramer) at Week 6 — 97.1 Percentage of participants
Transthyretin (TTR) Amyloid Cardiomyopathy
Primary: Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations — 8595; 5071 wins — p=0.0006
TTR-mediated Amyloidosis
Primary: The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation — 26; 7; 2 participants
ATTR-PN
Primary: Val30Met Group: Neuropathy Impairment Score Lower Limb (NIS-LL) Score at Baseline — 6.8; 11.6 units on a scale
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Primary: Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) — -21.345; -8.150 meters — p=0.0162
Amyloidosis
Primary: Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation — 49.0; 16.8; 0.0 percentage of participants
TTR-mediated Amyloidosis · Amyloidosis, Hereditary · Amyloid Neuropathies, Familial
Primary: Modified Neuropathy Impairment Score +7 (mNIS+7) — -6.03; 27.96 score on a scale — p=<0.0000001
FAP · Familial Amyloid Polyneuropathy · TTR
Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug — 100; 96.5; 36.0; 54.1 percentage of…
Transthyretin (TTR)-Mediated Amyloidosis · Familial Amyloidotic Polyneuropathy (FAP) · ATTR Amyloidosis
Primary: Percentage Change From Baseline in Serum TTR at Month 6 — -72.0 percentage change from baseline in TTR
Transthyretin Amyloid Cardiomyopathy
Primary: Number of Participants Who Experienced a Serious Adverse Event (SAE) — 0 Participants
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) · Amyloidosis, Hereditary · Amyloid Neuropathies, Familial
Primary: 6 Minute Walk Distance (6-MWD)
Transthyretin Amyloid Cardiomyopathy
Primary: Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) — 45; 21; 17; 6 Participants
FAP · Familial Amyloid Polyneuropathy · TTR
Primary: Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 — 4.16; 23.89 Scores on a Scale — p=0.00000004
Amyloidosis, Hereditary · Transthyretin Amyloidosis
Primary: Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group…
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
Primary: Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score — 2; 2; 3 Participants