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February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 6, 2020February 6, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 4, 2020February 4, 2020

Blood Levels of 3 Biomarkers May Help Identify Newborns at Risk for X-ALD, Chinese Study Suggests

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Measuring the levels of three biomarkers in the blood may help identify newborns at risk of X-linked adrenoleukodystrophy (X-ALD), a Chinese study suggests. The study, “Evaluation of a panel ... Read more

January 28, 2020January 28, 2020

European Medicines Agency Grants Orphan Drug Designation to Dimethyl Fumarate for ALD

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Dimethyl fumarate — an approved therapy for multiple sclerosis and psoriasis — has been granted orphan drug designation by the European Medicines Agency for the treatment of adrenoleukodystrophy (ALD). Orphan designation is ... Read more

January 21, 2020January 21, 2020

With Attention to Symptoms, Anesthesia Poses Low Risk of Complications for ALD Children, Study Finds

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If physicians pay close attention to the comorbidities associated with adrenoleukodystrophy (ALD) — particularly gastroesophageal reflux disease (GERD), adrenal insufficiency, and seizures — children with the disease may safely ... Read more

January 14, 2020January 14, 2020

Potential X-ALD Oral Therapy, Leriglitazone, Put on Fast Track by FDA

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Leriglitazone, a potential therapy for X-linked adrenoleukodystrophy (X-ALD), has been put on a fast track for review and possible approval by the U.S. Food and Drug Administration (FDA). Leriglitazone, ... Read more

January 7, 2020January 7, 2020

Spastic Paraparesia Can Be Only Symptom of Adrenomyeloneuropathy, Case Report Suggests

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Spastic paraparesia — a condition that involves weakness in the lower limbs and difficulty moving them — can be the only symptom of adult-onset adrenoleukodystrophy (ALD), a case report ... Read more

December 17, 2019December 17, 2019

Consider AMN Diagnosis in Men with Spastic Paraplegia Symptoms, Chinese Study Suggests

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Diagnosis of adrenomyeloneuropathy (AMN) — the adult-onset form of adrenoleukodystrophy (ALD) — should be considered in men with symptoms suggestive of hereditary spastic paraplegia, such as weakness in the legs, a Chinese ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

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