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When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.

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image: Exome Sequencing Helps Crack Rare Disease Diagnosis

Exome Sequencing Helps Crack Rare Disease Diagnosis

By Amanda B. Keener | May 1, 2018

Clinical analyses of patients’ gene sequences are helping to provide answers where none were available before.

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Crowdfunding can power investigations into diseases that would otherwise receive little attention.

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image: Slow March Toward a Canavan Cure

Slow March Toward a Canavan Cure

By Ashley Yeager | May 1, 2018

Two decades after a successful crowdfunding campaign, some clinical trial patients have seen improvements—but there’s still no approved treatment for the disease.

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The neuronal coverings that mediate synaptic changes are involved in everything from memory to psychiatric disorders, affecting autism, Alzheimer’s, and addiction.

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image: Eat Yourself to Live: Autophagy’s Role in Health and Disease

Eat Yourself to Live: Autophagy’s Role in Health and Disease

By Vikramjit Lahiri and Daniel J. Klionsky | March 1, 2018

New details of the molecular process by which our cells consume themselves point to therapeutic potential.

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image: Researchers Learn from Plant Viruses to Protect Crops

Researchers Learn from Plant Viruses to Protect Crops

By Claire Asher | February 1, 2018

Plants are locked in an ancient arms race with hostile viruses, but genome editing is giving crops the upper hand.

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image: Glial Ties to Persistent Pain

Glial Ties to Persistent Pain

By Mark R. Hutchinson | January 1, 2018

Immune-like cells in the central nervous system are now recognized as key participants in the creation and maintenance of persistent pain.

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image: Targeting Sodium Channels for Pain Relief

Targeting Sodium Channels for Pain Relief

By Catherine Offord | January 1, 2018

The race to develop analgesic drugs that inhibit sodium channel NaV1.7 is revealing a complex sensory role for the protein.

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New technologies reveal the dynamic changes in mouse and human embryos during the first week after fertilization.

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