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Adrenoleukodystrophy news

Adrenoleukodystrophy (ALD) Market Insights, Epidemiology and Market Forecast - 2027

Business Wire (press release) - May 31, 2018
One segment of the Adrenoleukodystrophy report encloses the detailed analysis of marketed drugs and late stage pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations ...

FDA Grants Breakthrough Therapy Designation to Gene Therapy for Cerebral Adrenoleukodystrophy

AJMC.com Managed Markets Network - Jun 3, 2018
The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc's Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy (CALD), an X-linked genetic disorder caused by a defect in the gene ABCD1. This mutation results in ...

Lenti-D Gets Breakthrough Tx Designation for Cerebral Adrenoleukodystrophy

Monthly Prescribing Reference - May 24, 2018
The Food and Drug Administration has granted Breakthrough Therapy designation to Lenti-D (bluebird bio) for the treatment of patients with cerebral adrenoleukodystrophy (CALD), also known as Lorenzo's Oil disease. Currently, the only treatment for CALD ...

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NEW Scientific Literature Review: X-linked Adrenoleukodystrophy by Mark Zaidi


X-Linked Adrenoleukodystrophy by Doriane Trompier (English) Paperback Book Free


X-linked Adrenoleukodystrophy (Colloquium Series on the Genetic Basis of Human


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Adrenoleukodystrophy videos

WHAT IS ALD? Adrenoleukodystrophy explained.

What is ALD? Adrenoleukodystrophy is a devastating genetic disease that affects the myelin around neurons in the brain and spinal cord. Learn the biology ...

Faces of Rare Disease Adrenoleukodystrophy YouTube


Deadly disease ravages boy’s brain

Just months after 5-year-old Carter Brischler was diagnosed with adrenoleukodystrophy (ALD), he lost the ability to see, hear, communicate and walk. Now, his ...

Adrenoleukodystrophy (ALD) mnemonic

ALD is a terrible disease that affects oxidation of the very long-chain fatty acids, so they accumulate in the brain, adrenal glands and the testes. It's an X-linked ...

ADRENOLEUKODYSTROPHY Symptoms and Treatments

Adrenoleukodystrophy (/-ˌlu-koʊ-ˈdis-trə-fiː/) (also known as X-linked adrenoleukodystrophy, ALD, X-ALD, adrenomyeloneuropathy, AMN, ...