News

April 21, 2020April 21, 2020

Stem Cell Transplant Safe, Effective in Halting Disease Progression in Adult-Onset Forms of ALD, Study Shows

News

Stem cell transplant is a safe and effective procedure to halt disease progression in patients with adult-onset forms of adrenoleukodystrophy (ALD), a study says. The study, “Clinical efficacy of haematopoietic ... Read more

April 14, 2020April 14, 2020

Spine’s Cross-Sectional Area May Capture Degree of Nerve Atrophy

News

The cross-sectional area (CSA) of the spinal cord in males with adrenoleukodystrophy (ALD) is smaller and flatter, and correlates with the severity of spinal cord degeneration (myelopathy) in those with ... Read more

April 7, 2020May 12, 2020

Bluebird to Request Lenti-D Approval for CALD in Europe, US Filing Delayed

News

Bluebird Bio remains on track to submit an application to the European Medicines Agency (EMA), requesting the approval of Lenti-D for boys with cerebral adrenoleukodystrophy (CALD) by year’s end. Due to the ... Read more

April 3, 2020April 3, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

April 1, 2020April 1, 2020

Overactive Bladder in Adrenoleukodystrophy and How to Deal With It

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Overactive bladder is a common problem in men with adrenomyeloneuropathy (AMN), a type of adrenoleukodystrophy (ALD), and also women who are carriers of the disease. What is overactive bladder? Overactive bladder is a ... Read more

March 31, 2020March 31, 2020

Stem Cell Treatment Fails to Help 2 Boys with Advanced Cerebral ALD

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An infusion of anti-inflammatory mesenchymal stem cells (MSC) given two boys with the cerebral form of adrenoleukodystrophy failed to halt or prevent disease progression, a case study reported. Whether the ... Read more

March 30, 2020March 30, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

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The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The ... Read more

March 25, 2020March 25, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

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Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the ... Read more

March 17, 2020March 17, 2020

MGTA-456 Stem Cell Therapy Leads to Lasting Neurological Benefits in CALD, Data Shows

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Magenta Therapeutics’ stem cell therapy MGTA-456 leads to clinically meaningful and lasting benefits in neurological and cognitive functions — as well as a resolution in brain inflammation — in children ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

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The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking ... Read more