7 Result(s)

Article

Efficacy and Safety of Sarilumab in patients with COVID19 Pneumonia: A Randomized, Phase III Clinical Trial (SARTRE Study)

SARS-CoV-2 pneumonia is often associated with hyper-inflammation. The cytokine-storm-like is one of the targets of current therapies for coronavirus disease 2019 (COVID-19). High Interleukin-6 (IL6) blood leve...

Aránzazu Sancho-López, Antonio F. Caballero-Bermejo… in Infectious Diseases and Therapy (2021)

Article

A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

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 tre...

Teresa Coelho, David Adams, Isabel Conceição… in Orphanet Journal of Rare Diseases (2020)

Article

Preliminary assessment of neuropathy progression in patients with hereditary ATTR amyloidosis after orthotopic liver transplantation (OLT)

David Adams, Juan Buades, Ole Suhr, Laura Obici… in Orphanet Journal of Rare Diseases (2015)

Article

When to decide to enroll a TTR-FAP patient in a Clinical Trial?

Juan Buades in Orphanet Journal of Rare Diseases (2015)

Article

Cardiac extracellular volume quantified with T1 map** techniques reflects degree of cardiac and neurological involvement in Hereditary Transthyretin Amyloidosis

Esther González-López, María Gallego-Delgado… in Orphanet Journal of Rare Diseases (2015)

Article

Phase 2 open-label extention (OLE) study of patisiran, an investigational siRNA agent for familial amyloidotic polyneuropathy (FAP)

David Adams, Ole B Suhr, Isabel Conceicao… in Orphanet Journal of Rare Diseases (2015)

Article

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharma...

Ole B Suhr, Teresa Coelho, Juan Buades, Jean Pouget… in Orphanet Journal of Rare Diseases (2015)