Gene Editing Could One Day Treat Muscle Disorders
Gene Editing Could One Day Treat Muscle Disorders
Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.
Gene Editing Could One Day Treat Muscle Disorders
Gene Editing Could One Day Treat Muscle Disorders

Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.

Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.

drug development
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Sandeep Ravindran | Aug 30, 2018
The disease is caused by mutations in a single gene. Can gene editing fix the problem?
Two CAR T-Cell Therapies Greenlighted in Europe
Two CAR T-Cell Therapies Greenlighted in Europe
Jef Akst | Aug 28, 2018
Cancer treatments from Novartis and Gilead earn approval from EU regulators, but a first pass by the UK’s state-funded health service finds CAR T is too expensive. 
Drug Approval Could Boost Research on Marijuana Treatment for Autism
Drug Approval Could Boost Research on Marijuana Treatment for Autism
Jessica Wright | Aug 17, 2018
Epidiolex, a cannabis-derived medicine for epilepsy, will prompt federal regulatory changes that could crack open access to study the plant.
The NIH Loosens Grip on Gene Therapy Trials
The NIH Loosens Grip on Gene Therapy Trials
Kerry Grens | Aug 16, 2018
The agency proposes ceding its scrutiny of these studies to the FDA.
First RNAi Therapy Approved by FDA
First RNAi Therapy Approved by FDA
Kerry Grens | Aug 10, 2018
Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.
Opinion: Learning from Immunotherapy’s Recent Failures
Opinion: Learning from Immunotherapy’s Recent Failures
Luis Felipe Campesato | Aug 1, 2018
The promise of immunotherapy is real. We now need to figure out how to maximize the number of patients the approach benefits.
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.
Immunotherapy More Effective in Men: Study
Immunotherapy More Effective in Men: Study
Kerry Grens | May 18, 2018
Women with metastatic cancer who were treated with a checkpoint inhibitor had a smaller benefit than did men.
FDA Approves Drug to Ease Symptoms of Opioid Withdrawal
FDA Approves Drug to Ease Symptoms of Opioid Withdrawal
Catherine Offord | May 17, 2018
Lucemyra is the first non-opioid drug to be approved in the U.S. specifically for this purpose.