10 documents found, page 1 of 1
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-me...
Adams, David; Tournev, Ivailo L.; Taylor, Mark S.; Coelho, Teresa; Planté-Bordeneuve, Violaine; Berk, John L.; González-Duarte, AlejandraBackground: The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy. Methods: HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were randomized 3:1 ...
Long-term efficacy and safety of inotersen for hereditary transthyretin amyloid...
Brannagan, Thomas H.; Coelho, Teresa; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Berk, John L.; Drachman, Brian; Gorevic, PeterBackground: Hereditary transthyretin amyloidosis (hATTR/ATTRv) results from the deposition of misfolded transthyretin (TTR) throughout the body, including peripheral nerves. Inotersen, an antisense oligonucleotide inhibitor of hepatic TTR production, demonstrated a favorable efficacy and safety profile in patients with the polyneuropathy associated with hATTR in the NEURO-TTR (NCT01737398) study. We report long...
Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense ...
Coelho, Teresa; Ando, Yukio; Benson, Merrill D.; Berk, John L.; Waddington-Cruz, Márcia; Dyck, Peter J.; Gillmore, Julian D.; Khella, Sami L.Introduction: AKCEA-TTR-LRx is a ligand-conjugated antisense (LICA) drug in development for the treatment of hereditary transthyretin amyloidosis (hATTR), a fatal disease caused by mutations in the transthyretin (TTR) gene. AKCEA-TTR-LRx shares the same nucleotide sequence as inotersen, an antisense medicine approved for use in hATTR polyneuropathy (hATTR-PN). Unlike inotersen, AKCEA-TTR-LRx is conjugated to a ...
ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical r...
Brannagan, Thomas H.; Auer-Grumbach, Michaela; Berk, John L.; Briani, Chiara; Bril, Vera; Coelho, Teresa; Damy, Thibaud; Dispenzieri, AngelaBackground: The global spread of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection causing the ongoing coronavirus disease 2019 (COVID-19) pandemic has raised serious concern for patients with chronic disease. A correlation has been identified between the severity of COVID-19 and a patient's preexisting comorbidities. Although COVID-19 primarily involves the respiratory system, dysfunction ...
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediate...
Adams, David; Polydefkis, Michael; González-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt V.; Schmidt, Hartmut H.; Berk, John L.Background: Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Methods: This multicentre, open-label ex...
Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin ...
Dyck, P. James B.; Coelho, Teresa; Waddington Cruz, Marcia; Brannagan, Thomas H.; Khella, Sami; Karam, Chafic; Berk, John L.; Polydefkis, Michael J.Introduction: Hereditary transthyretin-mediated amyloidosis (hATTR) manifests as multisystem dysfunction, including progressive polyneuropathy. Inotersen, an antisense oligonucleotide, improved the course of neuropathic impairment in patients with hATTR in the pivotal NEURO-TTR study (NCT01737398). To determine inotersen's impact on symptoms and patients' neuropathy experience, we performed a post hoc analysis ...
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therape...
González-Duarte, Alejandra; Berk, John L.; Quan, Dianna; Mauermann, Michelle L.; Schmidt, Hartmut H.; Polydefkis, Michael; Waddington-Cruz, MárciaHereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, debilitating disease often resulting in early-onset, life-impacting autonomic dysfunction. The effect of the RNAi therapeutic, patisiran, on autonomic neuropathy manifestations in patients with hATTR amyloidosis with polyneuropathy in the phase III APOLLO study is reported. Patients received patisiran 0.3 mg/kg intravenously (n = 148) or pl...
Inotersen preserves or improves quality of life in hereditary transthyretin amy...
Coelho, Teresa; Yarlas, Aaron; Waddington-Cruz, Marcia; White, Michelle K.; Sikora Kessler, Asia; Lovley, Andrew; Pollock, Michael; Guthrie, SpencerObjective: To examine the impact on quality of life (QOL) of patients with hATTR amyloidosis with polyneuropathy treated with inotersen (Tegsedi™) versus placebo. Methods: Data were from the NEURO-TTR trial (ClinicalTrials.gov Identifier: NCT01737398), a phase 3, multinational, randomized, double-blind, placebo-controlled study of inotersen in patients with hATTR amyloidosis with polyneuropathy. At baseline and...
A phase II, open-label, extension study of long-term patisiran treatment in pat...
Coelho, Teresa; Adams, David; Conceição, Isabel; Waddington-Cruz, Márcia; Schmidt, Hartmut H.; Buades, Juan; Campistol, Josep; Berk, John L.Background: Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to improve polyneuropathy and quality of life following 18 months of treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. In this 24-month Phase II open-label extension study, we evaluated the effects of patisiran treatment (0.3 mg/kg i...
A phase II, open-label, extension study of long-term patisiran treatment in pat...
Coelho, Teresa; Adams, David; Conceição, isabel; Waddington-Cruz, Márcia; Schmidt, Hartmut H.; Buades, Juan; Campistol, Josep; Berk, John L.Background: Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to improve polyneuropathy and quality of life following 18 months of treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. In this 24-month Phase II open-label extension study, we evaluated the effects of patisiran treatment (0.3 mg/kg i...