No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

Testing in animals and lab-grown cells suggests the experimental drug could enhance the function of lysosomes within cells.

The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.

As research and therapeutic applications for pluripotent stem cells (PSCs) grow, so does the need to reliably and cost-effectively generate large numbers of high-quality cells.

Screens of hundreds of drug excipients reveal that some can interact with biological targets, contradicting their FDA categorization as inert.

Frances Oldham Kelsey, a physician who halted use of a drug that caused birth defects in babies, has passed away at age 101.

Long-term marijuana smokers have less gray matter in their orbitofrontal cortex than nonsmokers, but other brain circuits may compensate by increasing connectivity.

Investigational drug that inhibits proteins involved with epigenetic regulation shows activity against certain blood cancers in an early-stage clinical trial.

Regulators must consider both the promise and potential pitfalls of new technologies when determining whether to move them into clinical trials.

A panel of advisors says that the agency should loosen restrictions on the sale of a diabetes drug with a spotty clinical safety record.
 

After numerous high-profile safety scares, clinicians and regulators push to fix critical weaknesses in the FDA’s monitoring system for approved drugs.

Patient safety is paramount in regulating new stem cell treatments.

Doctors question whether a government initiative to speed approval for promising new drugs is ensuring safety.

A consumer advocacy group calls for a ban of a diabetes drug after evidence surfaces that it may increase the risk of cancer.

The US regulatory agency recalled 54 percent more drugs from the second to third quarters of 2011.

Pharmacovigilance must keep pace with global distribution of drugs in resource-limited settings.

Europe’s anti-fraud office has launched an investigation into potential conflicts of interest at the EU’s drug approval agency.