Clinical Trials Save Lives and One Woman Wants in for Pompe Disease

In these times of political uncertainty, when budgets are being slashed, and medical programs are being defunded without regard to the people who will ultimately suffer, I certainly hope that the National Institutes of Health escapes the funding guillotine. I feel this way for a number of reasons, but first and foremost, the NIH are the folks that conduct clinical trials that produce new treatments for both common and rare diseases. This is a very big deal, and one of the freedoms we enjoy in the United States is the opportunity to participate in these trials. I personally know several people who are alive today because of their participation in clinical trials.

There is a young woman in India who, no doubt, agrees with my perspective. Meryl Mammen, 26, has Pompe disease and recently spoke out about the lack of clinical trials in her country. Pompe is a progressive neuromuscular disorder that occurs when the person is missing a certain enzyme.

Unfortunately for Mammen, the level of awareness about her disease is very low, and treatment, in the form of enzyme replacement therapy, was only introduced in India in 2010. She stated that while other countries are engaged in clinical trials for Pompe, those trials include people who are genetically different than the population in her native country. In the meanwhile, Mammen is taking the time to raise awareness on her own. We at Patient Worthy wish her well.

Erica Zahn

Erica Zahn

Erica Zahn is passionate about raising awareness of rare diseases and disorders and helping people connect with the resources that may ease their journey. Erica has been a caregiver, and is a patient, herself, so she completely relates to the rare disease community--on a deeply personal level.

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