Molecular Medicine Israel

Can Down Syndrome Be Treated?

People with Down syndrome are signing up for trials of several drugs aimed at helping their cognitive impairment, even as researchers explore more radical ideas to “cure” the genetic disorder

The cognitive impairments of Down syndrome, which is caused by an extra copy of chromosome 21, have long been considered untreatable, but recent advances in neuroscience and genetics are starting to raise the prospect of pharmaceutical interventions. Several clinical trials of drugs to improve memory and learning in adults with Down syndrome are now under way. If the trials succeed, the new drugs could help people with Down syndrome live more independently, but running the trials raises some logistical and ethical challenges, including recruiting enough participants and gaining their informed consent. As these trials move ahead, other labs are attempting to steer brain development onto a more typical path early on, for example, by inactivating the extra chromosome.

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