Vutrisiran — now known by the brand name Amvuttra — has been approved by the U.S. Food and Drug…
Articles by Marisa Wexler, MS
The European Commission is expected to propose a new governing framework for health data next month, called the European Health…
The U.S. Food and Drug Administration (FDA) is extending by three months its review timeline for vutrisiran, an experimental…
A clinical trial of the gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) is expanding to include those with…
Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such…
Note: This story was updated Nov. 24, 2021 to clarify that NTLA-2001 works by disrupting the TTR gene to reduce…
Tegsedi (inotersen), an approved treatment for familial amyloid polyneuropathy (FAP), has received a Category 1 classification by the…
Onpattro (patisiran) is now reimbursed for patients with familial amyloid polyneuropathy (FAP) living across most regions of Canada,…
Challenges with mental health often follow a person being told that they carry a mutation that causes familial amyloid…
Treatment with disease-modifying therapies can extend survival in people with familial amyloid polyneuropathy (FAP), a new study indicates. The…