News

August 10, 2020August 10, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

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 3, 2020August 19, 2020

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

News

To raise awareness of the rare disease communities and celebrate the creativity of its members, the EveryLife Foundation for Rare Diseases is seeking submissions to the 2020 Rare Artist contest. ... Read more

July 20, 2020July 20, 2020

AMN May Be Diagnosis for Patients With Spinal Cord Lesions, Case Report Says

News

Adrenomyeloneuropathy (AMN) should be considered in the differential diagnosis of patients showing signs of spinal cord lesions caused by the loss of myelin, the fatty sheath that wraps around and ... Read more

July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in ... Read more

June 29, 2020June 29, 2020

Certain Epigenetic Medicines May Help Compensate for ABCD1 Gene Deficiency, Study Suggests

News

Treatment with certain epigenetic medicines may help compensate for a deficiency in the ABCD1 gene in patients with adrenoleukodystrophy (ALD), a study suggests. Researchers found Merck’s epigenetic modifier Zolinza (vorinostat) ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support ... Read more

June 15, 2020June 15, 2020

Autobahn Raises $76M to Advance ABX-002 Candidate for CNS Disorders

News

Autobahn Therapeutics has raised $76 million in Series B funding to advance the development of ABX-002, the company’s lead therapeutic candidate for adrenomyeloneuropathy (AMN) and multiple sclerosis (MS). ABX-002 is ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for ... Read more

June 1, 2020June 1, 2020

ALD May Underlie Unexplained Adrenal Insufficiency in Boys, Case Report Shows

News

Boys with unexplained adrenal insufficiency should be screened for adrenoleukodystrophy (ALD), and repeated blood testing for very-long chain fatty acids (VLCFAs) may be key to its diagnosis, a case report suggests. ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies ... Read more