News

October 6, 2020October 6, 2020

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

News, syndicated

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 contest to heighten awareness of rare ... Read more

September 29, 2020September 30, 2020

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

News

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 ... Read more

September 22, 2020September 22, 2020

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

News

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 ... Read more

September 15, 2020September 15, 2020

Actin Protein Alterations May Underlie TTR-induced Neurodegeneration

News

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 ... Read more

September 8, 2020September 8, 2020

Alnylam Announces Potential New Biannual Dosing Regimen for Vutrisiran

News

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, ... Read more

September 2, 2020September 2, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 1, 2020September 1, 2020

Vyndaqel Remains Safe After 3 Years of Treatment, Analysis Shows

News

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 ... Read more

August 26, 2020August 26, 2020

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

News, syndicated

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 ... Read more

August 25, 2020August 25, 2020

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

News

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 ... Read more

August 20, 2020August 20, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

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. ... Read more