Adrenoleukodystrophy News

November 25, 2020November 26, 2020

EUnetHTA Seeks Patient, Caregiver Input for Joint Assessment on CALD Therapy

EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 and younger, with early cerebral adrenoleukodystrophy (CALD), for a new Joint Assessment on a medicinal ... Read more

November 25, 2020November 25, 2020

Tips for Dealing With Fatigue When You Have ALD

Adrenoleukodystrophy News

Fatigue is a common symptom for patients with chronic diseases such as adrenoleukodystrophy (ALD). Fatigue can be defined as extended periods of exhaustion leading to a lack of energy and ... Read more

November 18, 2020November 19, 2020

High Biotin Doses Restore Nerve Cell Health in ALD Mice, Study Shows

Adrenoleukodystrophy News

High doses of the B vitamin biotin restored metabolic balance and rescued nerve degradation in a mouse model of adrenoleukodystrophy (ALD), a study found. Based on the finding, the researchers ... Read more

November 11, 2020November 12, 2020

Trials Testing Leriglitazone Get $29.4M in Funding From European Investment Bank

Adrenoleukodystrophy News, COVID-19 News

Clinical trials testing Minoryx Therapeutics‘ leriglitazone (MIN-102), an investigational oral therapy for all forms of X-linked adrenoleukodystrophy (ALD) — including childhood cerebral ALD (CALD) and adrenomyeloneuropathy (AMN) — will receive ... Read more

November 11, 2020November 11, 2020

Medications With No Clear Benefit for Adrenoleukodystrophy

Adrenoleukodystrophy News

A number of medications have been proposed as treatment options for adrenoleukodystrophy (ALD) and have had promising starts. However, further investigation in clinical trials showed a lack of benefit or ... Read more

November 4, 2020November 4, 2020

Treatment Plan for Adrenoleukodystrophy

Adrenoleukodystrophy News

Adrenoleukodystrophy is a rare genetic brain disorder that has been categorized into three different forms: childhood cerebral adrenoleukodystrophy (CALD), adrenomyeloneuropathy (AMN), and Addison’s disease. If you or your child has ... Read more

October 28, 2020October 28, 2020

Planning for the Future When Your Child Has Adrenoleukodystrophy

Adrenoleukodystrophy News

You were likely prepared to hear your child was ill, but a diagnosis of a rare and progressive disorder like adrenoleukodystrophy (ALD) could still come as a shock. Among next steps you might ... Read more

October 21, 2020October 21, 2020

Just Diagnosed With Adrenoleukodystrophy, Now What?

Adrenoleukodystrophy News

A new diagnosis of a rare genetic disorder such as adrenoleukodystrophy (ALD) for your child or yourself can be frightening. Here is some information that may be helpful in understanding ... Read more

October 14, 2020October 15, 2020

Eli-cel Gene Therapy Continues to Show Encouraging Results for Boys with CALD, Clinical Data Show

Adrenoleukodystrophy News

Data from the Starbeam Phase 2/3 trial continues to support the safety and effectiveness of the gene therapy candidate elivaldogene autotemcel (eli-cel, formerly known as Lenti-D) for the treatment of cerebral adrenoleukodystrophy (CALD), Bluebird ... Read more

October 14, 2020October 14, 2020

Independent Living for Adults With Adrenoleukodystrophy

Adrenoleukodystrophy News

Having a chronic condition such as adrenoleukodystrophy (ALD) can be particularly concerning if you are living alone. But there are steps you can take to help you live well and independently. Learn about ... Read more

October 7, 2020October 8, 2020

Approval of CALD Gene Therapy, Lenti-D, Under Review in Europe

Adrenoleukodystrophy News

The European Medicines Agency (EMA) accepted an application requesting approval of the gene therapy Lenti-D (elivaldogene autotemcel, or eli-cel) to treat cerebral adrenoleukodystrophy (CALD), its developer, Bluebird Bio, announced. If approved, the submission — in the ... Read more

October 7, 2020October 7, 2020

Adrenoleukodystrophy and Sexuality

Adrenoleukodystrophy News

Adrenoleukodystrophy (ALD) and its adult-onset form, adrenomyeloneuropathy (AMN), can have both direct and indirect effects on a person’s sexuality. The following information may be useful as you manage this issue. ... Read more

September 30, 2020September 30, 2020

Adrenoleukodystrophy and Depression

Adrenoleukodystrophy News

Roughly 7% of adults experience depression every year in the U.S. People with adrenomyeloneuropathy (AMN), the adult-onset form of adrenoleukodystrophy (ALD) may be at a higher risk of depression or other ... Read more

September 23, 2020September 23, 2020

Phase 1 Study of VK0214 as Potential Oral Treatment for ALD to Open

Adrenoleukodystrophy News

Viking Therapeutics is launching a Phase 1 clinical trial testing increasing doses of VK0214, its oral investigative molecule to treat adrenoleukodystrophy (ALD), against a placebo in healthy people. “We are excited to ... Read more

September 23, 2020September 23, 2020

Warning Signs of Depression in Adrenoleukodystrophy Caregivers

Adrenoleukodystrophy News

It can be exhausting and stressful to be the parent or caregiver of someone who has a chronic disorder such as adrenoleukodystrophy. Because stress can trigger anxiety and depression, among other ... Read more