gene therapy, neuroscience, culture
Rare to the Rescue
Rare to the Rescue
Michael Yeaman, Victoria Jackson | May 1, 2018
Rarity is a strength, not a weakness, when lessons learned from rare disease patients buoy research and development to find cures for more common diseases.
Book Excerpt from <em>The Power of Rare</em>
Book Excerpt from The Power of Rare
Victoria Jackson, Michael Yeaman | May 1, 2018
In chapter 4, “Building a Cure Machine,” author Victoria Jackson reveals the challenges in launching a foundation focused on funding research on a rare disease.
Ali&rsquo;s Journey
Ali’s Journey
The Scientist Staff | May 1, 2018
Ali Guthy, the daughter of cosmetics entrepreneur Victoria Jackson, discusses NMO, the rare autoimmune disease she suffers from.
Families of Children with Rare Diseases Fuel Gene Therapy Research
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist Staff | May 1, 2018
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
Diana Kwon | May 1, 2018
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.
The Philadelphia Family Helping to Drive Research on MLD
The Philadelphia Family Helping to Drive Research on MLD
Catherine Offord | May 1, 2018
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.
Slow March Toward a Canavan Cure
Slow March Toward a Canavan Cure
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.
Infographic: Getting Synapses Ready to Fire
Infographic: Getting Synapses Ready to Fire
Ashley Yeager | May 1, 2018
A new study reveals more about the role of specialized Schwann cells at junctions between neurons and muscle cells.
Learning Enhances Synapses Between “Memory Cells” in Mice
Learning Enhances Synapses Between “Memory Cells” in Mice
Diana Kwon | Apr 26, 2018
A new technique reveals certain neuronal connections grow larger and denser when memories are made.
Worms’ Magnetic Sense Questioned
Worms’ Magnetic Sense Questioned
Abby Olena | Apr 25, 2018
Unsuccessful attempts to reproduce the results of a 2015 study reporting that C. elegans orient themselves by Earth’s magnetic field spark debate among researchers.