Posts by: Joana Carvalho

Joana Carvalho

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

October 27, 2020October 27, 2020

TTR Variant, E61K, That’s Not Prone to Clumping Seen to Cause FAP in Other Ways

News

Although less able to form damaging protein deposits, the E61K variant of transthyretin appears to cause familial amyloid polyneuropathy (FAP) through other mechanisms, a study suggested. The study, “A ... Read more

September 29, 2020September 30, 2020

Blood NfL Levels May Be Useful Marker of Polyneuropathy and Its Severity

News

Blood levels of neurofilament light chain (NfL) — a protein commonly used as a marker of nerve cell death in neurodegenerative disorders — associate with the severity of peripheral ... Read more

September 8, 2020September 8, 2020

Alnylam Announces Potential New Biannual Dosing Regimen for Vutrisiran

News

Alnylam Pharmaceuticals announced a potential new biannual dosing regimen for vutrisiran, its investigational treatment for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. In its “RNAi Roundtable” webinar, ... Read more

June 2, 2020June 2, 2020

Tegsedi Approved for Reimbursement in Portugal

News

The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP) ... Read more

May 19, 2020June 2, 2020

Tegsedi Available to FAP Patients in Spain at Reduced Cost

News

Spain’s Ministry of Health has added Tegsedi (inotersen), an injection treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP), to the national list of prescription medicines ... Read more

April 21, 2020April 21, 2020

FDA Places Vutrisiran on Fast Track as Potential FAP Treatment for Adults

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Alnylam Pharmaceuticals’ vutrisiran as a potential treatment of adults with familial amyloid polyneuropathy (FAP). Fast track is intended to ... Read more

April 14, 2020April 14, 2020

Sudoscan Test of Sweat Gland Function May Improve FAP Diagnosis, Study Says

News

Sudoscan, a device that evaluates sweat gland function, may help physicians improve the diagnosis of familial amyloid polyneuropathy — distinguishing these patients from those with chronic inflammatory demyelinating polyneuropathy, ... Read more

November 26, 2019November 26, 2019

Akcea and Ionis Launch Phase 3 Trial of AKCEA-TTR-LRx in People With FAP

News

Akcea Therapeutics and Ionis Pharmaceuticals announced the launch of a Phase 3 clinical trial evaluating the safety and efficacy of AKCEA-TTR-LRx (ION-682884) for the treatment of adults with familial ... Read more

September 17, 2019September 17, 2019

AKCEA-TTR-LRx Reduces Levels of TTR in Healthy Volunteers, Phase 1 Trial Shows

News

Akcea Therapeutics’ next-generation antisense medicine AKCEA-TTR-LRx (ION-682884) — currently in development for the treatment of all forms of familial amyloid polyneuropathy (FAP) — reduces the levels of a key ... Read more

August 13, 2019August 13, 2019

Tegsedi and Onpattro Effectively Stop FAP Progression, Improve Quality of Life, Review Suggests

News

Tegsedi (inotersen) and Onpattro (patisiran) are both effective treatments to stop the progression of familial amyloid polyneuropathy (FAP), alleviate gastrointestinal symptoms, and improve patients’ overall quality of life, a review ... Read more