Metachromatic Leukodystrophy
What is Metachromatic Leukodystrophy?
Metachromatic Leukodystrophy is a rare lysosomal storage disease.
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| Title | Date | Link |
|---|---|---|
| Testing for rare diseases at birth may have spared our girl a life of suffering, says mother of five-year-old who has degenerative brain condition | 03/04/2020 | |
| Girl with rare deadly disease receives revolutionary million-dollar gene-therapy drug – but it is too late for her older sister | 02/20/2023 | |
| Heartbreak of children lost to rare disease for which there was no cure | 03/04/2023 | |
| “A Second Chance at Life”: Can Gene Therapies Beat Rare Disease? | 04/01/2023 | |
| My daughter has a rare disease. We shouldn't have had to leave the US to save her life. | 03/09/2024 | |
| A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access | 03/21/2024 | |
| ‘There is no cure for this’: Rare disease causing 6-year-old girl to age rapidly | 06/28/2024 | |
| The most expensive drugs in the US | 08/29/2024 |
Clinical Trials
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access.
Enrolling is easy.
- Complete the screening form.
- Review the informed consent.
- Answer the permission and data sharing questions.
After these steps, the enrollment process is complete. All other questions are voluntary. However, these questions are important to patients and their families to create awareness as well as to researchers to study rare diseases. This is why we ask our participants to update their information annually or anytime changes to their information occur.
Researchers can contact CoRDS to determine if the registry contains participants with the rare disease they are researching. If the researcher determines there is a sufficient number of participants or data on the rare disease of interest within the registry, the researcher can apply for access. Upon approval from the CoRDS Scientific Advisory Board, CoRDS staff will reach out to participants on behalf of the researcher. It is then up to the participant to determine if they would like to join the study.
Visit sanfordresearch.org/CoRDS to enroll.
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