Why clinical trials are so important for the rare disease community.

Breaking down the differences between observational studies and clinical studies for drug research.

Ongoing clinical trials are paving the way for reducing steroid dependency and improving treatment options for various medical conditions.

The bittersweet and unpredictable chain of events that made Donna Rae Menard a believer in the clinical trial process.

For many of us in the rare disease community who have no approved treatments available, clinical trials are a powerful source of hope. Read on to learn about the benefits of joining a research study and how you can take part in one.

If you are interested in joining a research study, you may encounter some difficulties in navigating clinical trial listings. Here’s a quick guide to help you understand the terminology you might find in a clinical trial listing.

As a member of the rare community, you may know something about the process a new medication must go through before it receives FDA approval. Clinical trial phases 1 through 3 are conducted to establish the safety, effectiveness, and optimal dosage of a new treatment before it is made available to the public. But did you know that some drugs undergo an additional step post-approval to track their performance in the real world? These post-marketing studies, called phase 4 studies, help manufacturers learn more about long-term benefits and risks and offer a number of benefits to study participants.

7 tips for finding the right clinical trial for you and how Know Rare makes it easier to find a clinical study and talk to a researcher.

For many, “experimental” is what may come to mind when hearing about a clinical trial, which makes the prospect scary. However, for someone who has a rare disease without a treatment, the course ahead may already be filled with so many unknowns. Participating in a clinical trial can offer the opportunity to get access to care with doctors who are likely experts in the studied disease. But the key is, do not be afraid to ask.

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If you’ve spent any time reading up on or living life with a rare condition, chances are you’ve come across the terms “genetic disorder,” and “gene therapy.” But what exactly do they mean? And what differentiates them from other diseases and treatments?

Here’s a staggering statistic: only 5 percent of rare diseases have approved treatments. But that doesn’t mean there isn’t extensive work and research being done to find therapies, procedures, and eventually, cures for these conditions.