In late May, Novartis AG's Avexis Inc. unit gained FDA approval for Zolgensma (onasemnogene neparvovec) to treat spinal muscular atrophy, and other companies are looking to follow suit developing drugs to treat a variety of diseases of the central nervous system (CNS).

With the 2012 BIO International Convention behind us and the Fourth of July signaling summer vacation season in earnest, thoughts turn to visions of sun, sand and afternoons lounging in a deck chair. Again this year, BioWorld polled biotech execs, industry analysts and our own staff to construct a diverse list of titles for your reading pleasure. Whether your tastes run to historical intrigue or the classics, professional development or science fiction, you’ll find something of interest on our sixth annual list. Fiction: 16th Century England to 20th Century Los Angeles Diego Miralles, head of Janssen West Coast Research Center...

If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through. But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need to increase transparency and streamline its procedures was to set up an expert committee to investigate the activities and operations of its expert committees. I don’t imagine this is a cynical move by the recently installed head of EMA, Guido Rasi, to kick complaints about a lack of transparency and failure to listen to the needs of patients into the...

Reading last weekend’s Wall Street Journal review of “The Forever Fix: Gene Therapy and the Boy Who Saved It” I was struck by an anecdote. It’s about an interview the reviewer did with a scientist who works in the field of neuroprostheses, and that scientist’s refusal to talk about the possible practical applications of his work, because, he said, “false hope is a sinful thing.” Really? To me, it seems like an inevitable part of hope is that it might be false. To illustrate, I don’t hope that my neighbors will be nice to me, because it’s a sure thing....