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June 29, 2020June 29, 2020

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

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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 ... 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 ... Read more

June 15, 2020June 15, 2020

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

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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 ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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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 ... Read more

June 1, 2020June 1, 2020

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

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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 ... Read more

May 28, 2020May 28, 2020

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

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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 ... Read more

May 26, 2020May 26, 2020

ALD Testing Should Be Done in Males with Primary Adrenal Insufficiency of Unknown Cause, Case Report Contends

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Lack of testing for adrenoleukodystrophy (ALD) in males who have primary adrenal insufficiency due to an unknown cause may lead to a delayed or missing diagnosis of ALD. That ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 21, 2020May 21, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

News, syndicated

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

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