Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

gene therapy
New Treatments for Phenylketonuria Aim to Loosen Reins on Strict Diet
New Treatments for Phenylketonuria Aim to Loosen Reins on Strict Diet
Diana Kwon | May 29, 2018
Biotechs have developed enzyme replacements and genetically modified probiotics to treat patients with the rare metabolic disorder.
Patient Registries to Bolster Cell- and Gene-Therapy Clinical Trial Data
Patient Registries to Bolster Cell- and Gene-Therapy Clinical Trial Data
Anna Azvolinsky | May 15, 2018
As the first personalized cell and gene therapies are approved from small clinical trials, researchers propose the creation of publicly accessible databases to pull together real-world results.  
Biohacker Aaron Traywick Found Dead in DC Float Spa
Biohacker Aaron Traywick Found Dead in DC Float Spa
Ashley Yeager | May 1, 2018
The CEO of a biomedical startup had made headlines this year when he injected himself with an untested herpes vaccine in front of an audience.
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.
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.
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.
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.
Salk Institute Suspends Cancer Scientist Inder Verma
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
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.