News

December 18, 2020December 18, 2020

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

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The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, ... Read more

December 3, 2020December 3, 2020

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

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To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and ... Read more

December 2, 2020December 2, 2020

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

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Autobahn Therapeutics has successfully completed a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) regarding the ... Read more

November 26, 2020November 26, 2020

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

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The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, ... Read more

November 25, 2020November 25, 2020

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

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EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 and younger, with early cerebral adrenoleukodystrophy ... Read more

November 19, 2020November 19, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start ... Read more

November 18, 2020November 18, 2020

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

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

November 4, 2020November 4, 2020

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

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The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with ... Read more

October 28, 2020October 28, 2020

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

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A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured ... Read more