You can accelerate drug development in ALD

(adrenoleukodystrophy)

Your experience with ALD is important and can help researchers advance treatments for your community.

By joining AllStripes, you can contribute your de-identified medical records to multiple research efforts for ALD without leaving home. We do the work to collect your information, and you’ll receive all your records in your secure account, at no cost.

0/50 research participants

Help this community hit the research goal by sharing with your network!

How sharing your journey powers progress

  • Jumpstart new research for your rare disease

    Your (or your loved one’s) medical records contain clues that can lead to new treatments.

  • Determine important symptoms to target

    AllStripes researchers learn from patient records what would truly improve quality of life.

  • Speed up development of treatments

    Using cutting-edge tech we analyze your community’s data to improve trial design and chances of success.

  • Be part of multiple research efforts, without leaving home

    Your community’s de-identified records can advance multiple treatment studies.

BENEFITS TO JOINING

What you’ll get along the way

We do the work to collect all your records, at no cost, in your secure account. Access doctors’ notes you’ve never seen.

Access your records

We do the work to collect all your records, at no cost, in your secure account. Access doctors’ notes you’ve never seen.

See how your rare disease journey compares to others with your condition, and what’s helping.

Learn from your community

See how your rare disease journey compares to others with your condition, and what’s helping.

We’ll send you exclusive updates on the studies you’re contributing to and how you’re driving forward research.

Track your impact

We’ll send you exclusive updates on the studies you’re contributing to and how you’re driving forward research.

Join now

Our work with ALD research

ALD is a genetic condition caused by pathogenic variants in the ABCD1 gene, which lead to a buildup of a substance called very long chain fatty acids (VLCFAs) in tissues throughout the body, including in the brain. This buildup damages the protective layer of the nerve cells of the brain, impacting brain function and leading to progressive neurological symptoms which may include vision problems, deafness, difficulty swallowing, seizures, muscle weakness, and dementia. Most patients with ALD also have adrenal insufficiency, meaning their adrenal glands do not produce enough cortisol and aldosterone (hormones that help the body respond to stress and regulate blood pressure and water and salt balance). Patients with ALD range widely in the severity and age of onset of symptoms. The most severe form of ALD onsets in childhood with progressive neurological dysfunction and is fatal without intervention. Adrenomyeloneuropathy (AMN) is the name of the adult-onset form of the condition, which is milder and progresses more slowly.

The ABCD1 gene is on the X-chromosome. Females have two X chromosomes and males have one. Males are usually more severely impacted by ALD because, unlike females, they do not have a second working copy of the ABCD1 gene. While females are considered carriers of ALD, many develop AMN-like symptoms in adulthood. In addition to hormone replacement and supportive care, some ALD patients undergo stem cell transplantation. Gene therapy is also under investigation as a treatment for ALD.

The latest on our research: We are jumpstarting our ALD program to accelerate research on the disorder to ultimately improve treatment options. We welcome all patients and caregivers interested in participating and spreading the word to the ALD community.

  • Jumpstart
  • Accelerate
  • Insights

The ALD program is in the jumpstart stage.

How it works

Sign up

1. Sign up

Create your private account and review our research consent (takes about 10 minutes)

We get to work

2. We get to work

We do all the behind-the-scenes work to retrieve and process your records

Impact research

3. Impact research

Power multiple research projects at once by contributing your de-identified data

Still have questions?

Check out our FAQs

Our values & principles.

1
Patients own their data

We believe that the only way to keep data accessible to the researchers who need it is by enabling patients to own and control their own data. Patients decide for themselves whether they want to privately and securely contribute to research, rather than having the decision made on their behalf.

2
We protect patient privacy

We abide by a strict research consent and privacy policy. We only share de-identified data with researchers, foundations, and therapeutic companies with patients’ consent. Protecting the privacy of patients and ensuring the security of their information is our highest priority.

3
Researchers gain access

We aim to make useful data accessible to all researchers who have legitimate research questions or research programs. For academic research, we offer access without charge. We take research ethics seriously, and we have policies in place to ensure that research is conducted with integrity.

4
Inclusiveness in the community

We don’t like to take sides—we give all members of the community the option to get involved. Success in rare disease research takes a village and we always welcome additional collaborators who can advance and benefit from the mission.

5
Driving forward therapeutic development

Our primary mission is to accelerate drug development, so we prioritize generating insights specifically relevant to therapeutic research. We invest deeply in quality, security, and compliance to make this a reality.