FAP News Today

October 22, 2020October 23, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series ... Read more

October 20, 2020October 20, 2020

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

FAP News Today

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 products Regulatory Agency (MHRA). Intellia Therapeutics is ... Read more

October 13, 2020October 13, 2020

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

COVID-19 News, FAP News Today

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 the damaging transthyretin (TTR) protein for ... Read more

September 29, 2020September 29, 2020

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

FAP News Today

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 nerve ... Read more

September 22, 2020September 22, 2020

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

FAP News Today

CRX-1008, an investigational therapy also known as SOM0226 or tolcapone, is safe and well-tolerated by patients as a treatment for familial amyloid polyneuropathy (FAP), and can stabilize the TTR protein in ... Read more

September 15, 2020September 15, 2020

Actin Protein Alterations May Underlie TTR-induced Neurodegeneration

FAP News Today

The characteristic toxic buildup of mutant transthyretin (TTR) protein clumps in familial amyloid polyneuropathy (FAP) affects the development of nerve cell fibers by interacting with regulators of a protein called ... Read more

September 8, 2020September 8, 2020

Alnylam Announces Potential New Biannual Dosing Regimen for Vutrisiran

FAP News Today

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, the ... Read more

September 1, 2020September 1, 2020

Vyndaqel Remains Safe After 3 Years of Treatment

FAP News Today

Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety concerns raised, according to an early analysis ... Read more

August 26, 2020September 28, 2020

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

FAP News Today

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 25, 2020August 26, 2020

Ultrasound Measurements of Nerve Size Could Help Diagnose FAP

FAP News Today

Using ultrasound to measure the size of the median nerve — which runs down the upper arm and continues into the forearm and hand — could be useful for the ... Read more

August 20, 2020September 20, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

FAP News Today

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 18, 2020August 18, 2020

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

FAP News Today

Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from the NEURO-TTR Phase 2/3 trial show. The findings, “Neuropathy Symptom and Change: ... Read more

August 10, 2020September 11, 2020

New Streaming Channel Showcases Rare Disease Films

COVID-19 News, FAP News Today

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

August 4, 2020August 4, 2020

Tegsedi Eases Disease Progression in FAP Patients, Trial Data Show

FAP News Today

Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show. The findings may be useful in designing ... Read more

July 21, 2020July 21, 2020

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

FAP News Today

Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. The ... Read more