Opinion: Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases

In addition to the most visible effects of Covid-19 — tragic deaths, lingering illness, economic turmoil, and more — the pandemic is affecting much that is out of the spotlight. One is research on the so-called rare and ultra-rare diseases that affect an estimated 300 million people around the world.

One of us (S.K.R.) has a 2-year-old boy, Raghav, with an ultra-rare disease. He is just one of nine kids in the world identified so far with a disease caused by a mutation in the glutathione peroxidase 4 (GPX4) gene. It causes extremely low muscle tone, difficulty eating, problems seeing and hearing, and abnormalities in the spine, pelvis, and long bones. But Raghav is lucky: Most babies born with this disease die shortly after birth.

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