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March 26, 2019March 26, 2019

20-Year Survival Seen in 73% of ALD and Other Metabolic Disorder Patients After Transplant, Study Reports

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Twenty years after a blood or marrow transplant (BMT), patients with adrenoleukodystrophy (ALD) and other metabolic disorders can have a greater than 70% survival rate, according to a new study. ... Read more

March 22, 2019March 22, 2019

Blood Stem Cell Therapy MGTA-456 Showing Signs of Efficacy in Cerebral ALD, Early Trial Data Show

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Magenta Therapeutics’ allogeneic blood stem cell therapy resolved brain inflammation and stopped the loss of white brain matter in two cerebral adrenoleukodystrophy (ALD) patients, early data from a Phase 2 clinical trial ... Read more

March 19, 2019March 19, 2019

Blood Stem Cell Transplant May Slow Cognitive Decline in Adult Cerebral ALD

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A blood stem cell transplant may halt disease progression in certain adults with cerebral adrenoleukodystrophy (ALD), a study reports. The study, “Allogeneic hematopoietic stem cell transplantation with myeloablative conditioning for adult ... Read more

March 15, 2019March 15, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this ... Read more

March 12, 2019March 12, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to ... Read more

March 5, 2019March 5, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to ... Read more

February 26, 2019February 26, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already ... Read more

February 22, 2019February 22, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March ... Read more

February 15, 2019February 15, 2019

More Sensitive Tools Needed to Assess ALD Spinal Cord Disease Progression in Women, Study Contends

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Comparison between approaches to assess spinal cord disease show that the neurological tool Expanded Disability Status Scale (EDSS) can assess disease progression in women with adrenoleukodystrophy (ALD), but only when ... Read more

February 12, 2019February 12, 2019

Benefits of X-ALD Newborn Screening in U.K. Outweigh Costs Incurred, Study Shows

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Including X-linked adrenoleukodystrophy (X-ALD) in newborn screening programs is cost-effective, because early diagnosis lowers care and education costs, and improves the chances of patient survival, researchers from the U.K. report. ... Read more