Why are natural history studies important?

Before a treatment can be tested in a clinical trial, researchers need to understand what happens to patients without treatment. Natural history studies provide this baseline. Participating in one is one of the most valuable contributions a patient can make to advancing research, even when no experimental therapy is involved. If you are not sure where to start, contact us and we can we can help direct you to local natural history studies.

Why is there currently no cure for LAMA2-RD? What research studies are currently underway? How long does it take to develop a new treatment? What are the stages of drug development? What is a natural history study? What is a patient registry? Why should I participate in a patient registry? Which universities and institutions are researching LAMA2-RD? Which companies are developing therapies? What are the biggest scientific challenges? Which treatment approaches are being investigated? What is gene therapy? What is CRISPR gene editing? What are stem cell therapies? What are antisense therapies?

Why are natural history studies important?

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