News

December 18, 2020December 18, 2020

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

News

The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, while raising awareness through their work of these ... Read more

December 3, 2020December 3, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

December 2, 2020December 2, 2020

Autobahn Plans to Start Clinical Development of AMN Candidate ABX-002 in 2021

News

Autobahn Therapeutics has successfully completed a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) regarding the development plan for its therapy ABX-002, a thyroid ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 25, 2020November 25, 2020

EUnetHTA Seeks Patient, Caregiver Input for Joint Assessment on CALD Therapy

News

EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 and younger, with early cerebral adrenoleukodystrophy (CALD), for a new Joint Assessment on a ... Read more

November 19, 2020November 19, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 18, 2020November 18, 2020

High Biotin Doses Restore Nerve Cell Health in ALD Mice, Study Shows

News

High doses of the B vitamin biotin restored metabolic balance and rescued nerve degradation in a mouse model of adrenoleukodystrophy (ALD), a study found. Based on the finding, the ... Read more

November 11, 2020November 11, 2020

Trials Testing Leriglitazone Get $29.4M in Funding From European Investment Bank

News

Clinical trials testing Minoryx Therapeutics‘ leriglitazone (MIN-102), an investigational oral therapy for all forms of X-linked adrenoleukodystrophy (ALD) — including childhood cerebral ALD (CALD) and adrenomyeloneuropathy (AMN) — will ... Read more

November 4, 2020November 4, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

October 28, 2020October 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News, syndicated

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more