Margarida Maia, PhD,  science writer—

Margarida is a biochemist (University of Porto, Portugal) with a PhD in biomedical sciences (VIB and KULeuven, Belgium). Her main interest is science communication. She is also passionate about design and the dialogue between art and science.

Articles by Margarida Maia

Family members of late-onset FAP patients face unique challenges

Family members of people with late-onset familial amyloid polyneuropathy (FAP) may face emotional and practical challenges due to the onset of disease symptoms in late adulthood, with some seeking information and support while others avoiding discussing the disease. That’s according to interviews of eight adults who were family members…

Tafamidis meglumine safely slows FAP progression: Brazil study

Tafamidis meglumine safely and effectively slows disease progression in most people living with familial amyloid polyneuropathy (FAP), according to a real-world study from Brazil. The findings were in line with data from clinical trials. “The efficacy and safety of tafamidis reported in clinical trials is expandable to the…

1st FAP patient dosed in Phase 3 trial of gene therapy nex-z

The first adult with familial amyloid polyneuropathy (FAP) has been dosed in a Phase 3 clinical trial testing nexiguran ziclumeran (nex-z), Intellia Therapeutics’ one-time gene therapy. The trial, called MAGNITUDE-2 (NCT06672237), is recruiting up to 50 adults diagnosed with FAP, also known as hereditary transthyretin (ATTR) amyloidosis…

ART001 gene editor named orphan drug for ATTR amyloidosis

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ART001, a gene editing candidate that Accuredit Therapeutics is developing as a one-time treatment for transthyretin (ATTR) amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP). Such a designation supports developing possible treatment…

Amvuttra approved in US to treat adults with ATTR-CM

The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary transthyretin amyloidosis (ATTR-CM). Alnylam Pharmaceuticals’ Amvuttra was specifically cleared for use to reduce death, hospital stays, and urgent visits due to heart problems in…