Updates

April 7, 2020April 7, 2020
Bluebird to Request Lenti-D Approval for CALD in Europe, US Filing Delayed

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) ... Read more
April 7, 2020April 8, 2020
Schooling Your Child With Adrenoleukodystrophy

If you are the parent of a child with a rare disease such as adrenoleukodystrophy (ALD), managing your child’s schooling and their disease can ... Read more
April 3, 2020April 3, 2020
NORD’s Advice on COVID-19: You’re Stronger Than You Think

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 ... Read more
April 1, 2020April 1, 2020
Overactive Bladder in Adrenoleukodystrophy and How to Deal With It

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 ... Read more
March 31, 2020March 31, 2020
Stem Cell Treatment Fails to Help 2 Boys with Advanced Cerebral ALD

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, ... Read more

News

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
March 30, 2020March 30, 2020
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 ...
News

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
March 25, 2020March 25, 2020
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate ...
News

MGTA-456 Stem Cell Therapy Leads to Lasting Neurological Benefits in CALD, Data Shows
March 17, 2020March 17, 2020
Magenta Therapeutics’ stem cell therapy MGTA-456 leads to clinically meaningful and lasting benefits in ...
Health Insight

Dealing with Bowel Dysfunction in Adrenoleukodystrophy
March 12, 2020March 13, 2020
Bowel dysfunction is the term given to problems with passing stool, which can lead ...
News

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
March 10, 2020March 10, 2020
The number of treatments for children with rare diseases has grown over the past decade, ...
News

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
March 5, 2020March 5, 2020
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
March 3, 2020March 3, 2020
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review ...
Dealing With Myelopathy in Adrenoleukodystrophy
February 27, 2020February 28, 2020
Adrenoleukodystrophy (ALD) is a rare neurological disorder characterized by the accumulation of saturated very-long-chain ...
New Worm Model May Help in More Quickly Finding Ways to Treat X-ALD, Study Shows
February 25, 2020February 25, 2020
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
February 20, 2020March 3, 2020
Dealing with Peripheral Neuropathy in Adrenoleukodystrophy
February 20, 2020February 21, 2020
Researchers Develop Simpler, Faster Method to Screen Newborns for X-ALD
February 18, 2020February 25, 2020

Tips for Taking Care of Adrenoleukodystrophy Caregivers
February 13, 2020February 14, 2020
Caregiving is a demanding task regardless of a person’s disorder. But with rare diseases ...
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
February 11, 2020February 11, 2020
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
February 6, 2020February 6, 2020
Support Groups for People Affected by Adrenoleukodystrophy
February 6, 2020February 7, 2020

Blood Levels of 3 Biomarkers May Help Identify Newborns at Risk for X-ALD, Chinese Study Suggests
February 4, 2020February 4, 2020
Measuring the levels of three biomarkers in the blood may help identify newborns at ...
Tips for Caregivers of Patients with Adrenoleukodystrophy
January 30, 2020January 31, 2020
European Medicines Agency Grants Orphan Drug Designation to Dimethyl Fumarate for ALD
January 28, 2020January 28, 2020

Health Insight

Tips to Keep Stress Levels Low When You Have Adrenoleukodystrophy
January 23, 2020January 24, 2020
Adrenoleukodystrophy (ALD) is a rare, genetic disease characterized by the accumulation of saturated very ...
News

With Attention to Symptoms, Anesthesia Poses Low Risk of Complications for ALD Children, Study Finds
January 21, 2020January 21, 2020
If physicians pay close attention to the comorbidities associated with adrenoleukodystrophy (ALD) — particularly ...
Health Insight

Tips for Maintaining a Personal Health Record When You Have Adrenoleukodystrophy
January 16, 2020January 17, 2020
Adrenoleukodystrophy (ALD) is a rare disease characterized by the accumulation of saturated very-long-chain fatty ...
News

Potential X-ALD Oral Therapy, Leriglitazone, Put on Fast Track by FDA
January 14, 2020January 14, 2020
Leriglitazone, a potential therapy for X-linked adrenoleukodystrophy (X-ALD), has been put on a fast ...
Health Insight

Adrenoleukodystrophy and Lorenzo’s Oil – What to Keep in Mind
January 9, 2020January 10, 2020
Lorenzo’s oil is a 4:1 mixture of two unsaturated fatty acids called oleic acid ...
News

Spastic Paraparesia Can Be Only Symptom of Adrenomyeloneuropathy, Case Report Suggests
January 7, 2020January 7, 2020
Spastic paraparesia — a condition that involves weakness in the lower limbs and difficulty ...
News

Consider AMN Diagnosis in Men with Spastic Paraplegia Symptoms, Chinese Study Suggests
December 17, 2019December 17, 2019
Diagnosis of adrenomyeloneuropathy (AMN) — the adult-onset form of adrenoleukodystrophy (ALD) — should be considered in men ...
News

Gene Therapy Takes Center Stage at 2019 NORD Summit
December 10, 2019December 10, 2019
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic ...
News

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
December 5, 2019
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or ...

Subscribe to our mailing list

Pin It on Pinterest