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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.
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.
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.
Families of Children with Rare Diseases Fuel Gene Therapy Research
Staff | May 1, 2018
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
Salk Institute Suspends Cancer Scientist Inder Verma
Ashley Yeager | Apr 23, 2018
The prominent researcher has been put on administrative leave pending an investigation into unspecified allegations.
World’s Largest Cell and Gene Therapy Plant Opens
Catherine Offord | Apr 11, 2018
Lonza will employ more than 200 full-time staff to work at the Texas-based facility, the company says.
Vision Restored: The Latest Technologies to Improve Sight
Anna Azvolinsky | Apr 10, 2018
Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness.
Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals
Jim Daley | Jan 31, 2018
Biotech stocks fell in response to the news.
Sanofi and Celgene Spend $20 Billion in Major Biotech Acquisitions
Katarina Zimmer | Jan 23, 2018
Pharmaceutical giant Sanofi to take over hemophilia drugmaker Biovertiv, while Celgene will buy cancer drugmaker Juno Therapeutics.
Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice
Shawna Williams | Jan 4, 2018
Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.