Updates

October 20, 2020October 20, 2020
UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

A Phase 1 trial of NTLA-2001, a potential gene editing therapy for familial amyloid polyneuropathy (FAP), was approved to open by the United Kingdom’s Medicines and Healthcare ... Read more
October 15, 2020October 15, 2020
FAQs About Kidney Transplants for FAP

Familial amyloid polyneuropathy (FAP) is a rare genetic disease that results in the buildup of amyloid fibrils, or protein clumps, in organs that include ... Read more
October 13, 2020October 13, 2020
After Promising Early Data on Gene Editing, Intellia Will Launch ATTR Trial This Year

A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene editing therapy for transthyretin amyloidosis (ATTR), results in a sustained reduction in the levels of ... Read more
October 8, 2020October 8, 2020
FAP and Your Kidneys

People with familial amyloid polyneuropathy (FAP) may well have problems with their kidneys. Without treatment, these can lead to serious complications. How does FAP ... Read more
October 6, 2020October 6, 2020
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar ... Read more

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September 6, 2017September 6, 2017

Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests

News

An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for ... Read more

Updates

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

FAQs About Kidney Transplants for FAP

After Promising Early Data on Gene Editing, Intellia Will Launch ATTR Trial This Year

FAP and Your Kidneys

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Subscribe to our mailing list

Magnetic Resonance Imaging (MRI) in FAP: What Does It Involve?

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

FAQs About Liver Transplant for FAP

Trial Data Provide Proof-of-concept for CRX-1008 as Leptomeningeal FAP Treatment

Digestive Problems in FAP – How Do You Deal With Them?

Actin Protein Alterations May Underlie TTR-induced Neurodegeneration

Tips for Managing Fatigue When You Have FAP

Alnylam Announces Potential New Biannual Dosing Regimen for Vutrisiran

Same But Different Photo Contest Celebrates People With Rare Diseases

Vyndaqel Remains Safe After 3 Years of Treatment, Analysis Shows

Tissue Biopsy for FAP: What to Expect

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Ultrasound Measurements of Nerve Size Could Help Diagnose FAP, Study Says

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Treatment Plan for FAP

Tegsedi Seen to Ease Neuropathy Symptoms for FAP Patients in Clinical Trial

How Age of Onset Affects Disease Progression in FAP

New Streaming Channel Showcases Rare Disease Films

FAP and Your Heart

Tegsedi Eases Disease Progression in FAP Patients, Trial Data Show

FAP and Your Eyes

Keeping a Food Diary When You Have FAP

Long-term Onpattro Use Safely Halts FAP Progression, Phase 2 Data Show

FAP and Diet

New Nerve Imaging Tool May Aid Early FAP Diagnosis, Study Suggests

Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests

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