Posts by: Magdalena Kegel

Magdalena Kegel

Magdalena is a writer with a passion for bridging the gap between the people performing research, and those who want or need to understand it. She writes about medical science and drug discovery. She holds an MS in Pharmaceutical Bioscience and a PhD — spanning the fields of psychiatry, immunology, and neuropharmacology — from Karolinska Institutet in Sweden.

January 16, 2018

Vyndaqel May Not Protect FAP Patients Against Anemia, Study Finds

News

While Vyndaqel (tafamidis) has been shown to improve symptoms of familial amyloid polyneuropathy (FAP), it likely does not protect against anemia — a common problem among patients with the disease, a ... Read more

January 11, 2018

Alnylam Continuing FAP Treatment Development Without Former Partner Sanofi

News

Alnylam Pharmaceuticals and Sanofi have dissolved the partnership they formed to develop and market two treatments for the neurological disease familial amyloid polyneuropathy, or FAP. The change is aimed at streamlining the development ... Read more

January 9, 2018

Inotersen a Step Closer to Approval as FDA Grants Priority Review to Ionis’ NDA

News

The U.S. Food and Drug Administration (FDA) has agreed to speedily review Ionis Pharmaceuticals’ application to get its investigational drug inotersen approved as a treatment for hereditary TTR amyloidosis (hATTR). ... Read more

December 21, 2017December 21, 2017

Prevalence Study Finds FAP May Be More Common Worldwide Than Previously Thought

News

An assessment of the global prevalence of familial amyloid polyneuropathy, caused by mutations in the transthyretin (TTR) gene, gave a similar count to that estimated in earlier studies. But ... Read more

December 19, 2017

Alnylam and Sanofi Ask European Regulators to Approve Patisiran for FAP

News

Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran for adults with hereditary transthyretin-mediated (hATTR) amyloidosis. The European Medicines Agency ... Read more

December 14, 2017December 19, 2017

Complete New Drug Application for FAP Therapy Patisiran Submitted to FDA

News

Alnylam Pharmaceuticals has submitted a complete new drug application (NDA) for its investigational therapy patisiran to the U.S. Food and Drug Administration for the treatment of adults with hereditary ... Read more

December 7, 2017

Cognitive Impairment Common Among Older, Untreated FAP Patients

News

Researchers have found that a large proportion of untreated people with the TTRVal30Met mutation — causing familial amyloid polyneuropathy (FAP) — have cognitive impairment, particularly if they experience disease symptoms. Such ... Read more

November 21, 2017

Alnylam Starts Rolling Submission of Application Asking FDA to Approve Patisiran

News

Alnylam Pharmaceuticals has started its submission of a rolling new drug application (NDA), asking the U.S. Food and Drug Administration (FDA) to approve patisiran for the treatment of hereditary ... Read more

November 6, 2017November 6, 2017

Alnylam Set to Seek Regulatory Approval for Patisiran With Robust Findings in Phase 3 FAP Trial

News

Alnylam Pharmaceuticals, together with its partner Sanofi Genzyme, has now released the full results from the Phase 3 trial of patisiran — a drug intended to treat hereditary ATTR ... Read more

October 31, 2017October 31, 2017

FAP Patients Have a Moderate Chance of Having Unaffected Children, Study Suggests

News

Patients with familial amyloid polyneuropathy (FAP) appear to have intact reproductive capacity, even when they have undergone a liver transplant or receive treatment with Vyndaqel (tafamidis), a retrospective study ... Read more