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

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Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash.

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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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Though Calliope Joy’s disease is too far progressed to be treatable, her parents have helped other children with metachromatic leukodystrophy get access to an experimental therapy.

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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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Multidrug combinations lead to better results for cancer patients, but efficiently identifying them is proving difficult.

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Research into the biological basis of gender identity is in its infancy, but clues are beginning to emerge.

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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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