A clinical trial of the gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) is expanding to include those with…
Articles by Marisa Horak, MS
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…
Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is…
Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…