Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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Dealing with Peripheral Neuropathy in Adrenoleukodystrophy
Peripheral neuropathy — damage to the peripheral nerves characterized by a feeling of tingling and pain in the hands and feet — is a ... Read more -
Researchers Develop Simpler, Faster Method to Screen Newborns for X-ALD
Researchers have developed a simpler, faster, reliable method of screening newborns for X-linked adrenoleukodystrophy (X-ALD), which was validated in a North Carolina pilot study. The work ... Read more -
Tips for Taking Care of Adrenoleukodystrophy Caregivers
Caregiving is a demanding task regardless of a person’s disorder. But with rare diseases such as adrenoleukodystrophy (ALD) it can be particularly challenging, given this ... Read more -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
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. ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ... -
Support Groups for People Affected by Adrenoleukodystrophy
Adrenoleukodystrophy (ALD) is a rare genetic disorder caused by mutations in the ABCD1 gene ... -
Blood Levels of 3 Biomarkers May Help Identify Newborns at Risk for X-ALD, Chinese Study Suggests
Measuring the levels of three biomarkers in the blood may help identify newborns at ... -
Tips for Caregivers of Patients with Adrenoleukodystrophy
Caring for someone with a rare disease such as adrenoleukodystrophy (ALD) can be very ... -
European Medicines Agency Grants Orphan Drug Designation to Dimethyl Fumarate for ALD
Dimethyl fumarate — an approved therapy for multiple sclerosis and psoriasis — has been ...
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