News

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 ... 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

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 ... 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

News, syndicated

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

May 19, 2020May 19, 2020

First Patient Dosed in NEXUS Phase 2 Trial of Leriglitazone in CALD

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A Phase 2 trial evaluating leriglitazone, Minoryx Therapeutics‘ investigational oral therapy for boys with cerebral adrenoleukodystrophy (CALD), has dosed its first patient at Hospital Sant Joan de Déu in Spain, the company ... Read more

May 12, 2020May 12, 2020

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

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Hitachi Chemical Advanced Therapeutics Solutions and Apceth Biopharma GmbH, both subsidiaries of Hitachi Chemical Company, recently signed a new long-term development and manufacturing agreement with Bluebird Bio. The agreement will ... Read more

May 5, 2020May 5, 2020

SwanBio Raises $52M to Support Studies of Gene Therapy for Adult-onset ALD

News

SwanBio Therapeutics announced that it raised $52 million to help advance its viral-based gene therapy for adult-onset adrenoleukodystrophy (ALD) in studies essential for clinical testing, and to expand the company’s portfolio ... Read more

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