Updates

June 1, 2020June 1, 2020
ALD May Underlie Unexplained Adrenal Insufficiency in Boys, Case Report Shows

Boys with unexplained adrenal insufficiency should be screened for adrenoleukodystrophy (ALD), and repeated blood testing for very-long chain fatty acids (VLCFAs) may be key to ... Read more
June 1, 2020June 3, 2020
Adrenoleukodystrophy and Vision

Adrenoleukodystrophy (ALD) is a rare genetic disease that can lead to neurodegeneration (nerve cell death) and adrenal insufficiency, among other symptoms. However, symptoms vary widely ... Read more
May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 26, 2020May 26, 2020
ALD Testing Should Be Done in Males with Primary Adrenal Insufficiency of Unknown Cause, Case Report Contends

Lack of testing for adrenoleukodystrophy (ALD) in males who have primary adrenal insufficiency due to an unknown cause may lead to a delayed or ... Read more
May 26, 2020May 27, 2020
Seizures in Adrenoleukodystrophy and How to Deal with Them

Adrenoleukodystrophy (ALD) is a rare genetic disease characterized by the buildup of saturated very-long-chain fatty acids (VLCFAs) inside cells. When the disease affects the ... Read more

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Updates

ALD May Underlie Unexplained Adrenal Insufficiency in Boys, Case Report Shows

Adrenoleukodystrophy and Vision

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

ALD Testing Should Be Done in Males with Primary Adrenal Insufficiency of Unknown Cause, Case Report Contends

Seizures in Adrenoleukodystrophy and How to Deal with Them

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

First Patient Dosed in NEXUS Phase 2 Trial of Leriglitazone in CALD

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

Adrenoleukodystrophy: What Are the First Signs?

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SwanBio Raises $52M to Support Studies of Gene Therapy for Adult-onset ALD

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Case Report Describes Novel ALD-causing Gene Mutation

What Is Newborn Screening and How Can It Help Identify Adrenoleukodystrophy?

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

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

Speech Therapy for Children with Adrenoleukodystrophy

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

Tips for Dealing with Hearing Loss in Adrenoleukodystrophy

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

Schooling Your Child With Adrenoleukodystrophy

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

Overactive Bladder in Adrenoleukodystrophy and How to Deal With It

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

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

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

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

Dealing with Bowel Dysfunction in Adrenoleukodystrophy

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

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ALD May Underlie Unexplained Adrenal Insufficiency in Boys, Case Report Shows

Adrenoleukodystrophy and Vision

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

ALD Testing Should Be Done in Males with Primary Adrenal Insufficiency of Unknown Cause, Case Report Contends

Seizures in Adrenoleukodystrophy and How to Deal with Them

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

First Patient Dosed in NEXUS Phase 2 Trial of Leriglitazone in CALD

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

Adrenoleukodystrophy: What Are the First Signs?

Subscribe to our mailing list

SwanBio Raises $52M to Support Studies of Gene Therapy for Adult-onset ALD

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Case Report Describes Novel ALD-causing Gene Mutation

What Is Newborn Screening and How Can It Help Identify Adrenoleukodystrophy?

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

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

Speech Therapy for Children with Adrenoleukodystrophy

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

Tips for Dealing with Hearing Loss in Adrenoleukodystrophy

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

Schooling Your Child With Adrenoleukodystrophy

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

Overactive Bladder in Adrenoleukodystrophy and How to Deal With It

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

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

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

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

Dealing with Bowel Dysfunction in Adrenoleukodystrophy

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

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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