Stunning Breakthrough: Hemgenix
Last week we shared an essay by Paul Clement about the approval for gene therapy for hemophilia A, approved only in Europe, and asked, when for the US? While that question is still valid for hemophilia A, the stunning news this week was that gene therapy–at long last—is approved, for hemophilia B! The news straight
Hemophilia Gene Therapy—is the US Next?
Paul Clement Last week we discussed Roctavian, the brand name of BioMarin Pharmaceutical’s gene therapy product, valoctocogene roxaparvovec, to treat patients with severe hemophilia A, and the European Commission’s August 24, 2022, conditional marketing authorization for the therapy. Two big questions on everyone’s mind are: when will it be approved in the US and what
Hemophilia Gene Therapy wins Conditional Approval
Part 1 Paul Clement It has been almost 30 years since the New York Times in 1994 ran the headline “Cure for Hemophilia Is Seen by Year 2000,” regarding a prediction by the World Health Organization. But the first approval of a gene therapy for hemophilia has finally arrived! In June, the European Medicines Agency
Hemo Nonprofit? Beware!
We had a real scare the week of Halloween… cyber thieves had raided our corporate bank account and were draining it. Luckily my bookkeeper and I noticed some odd and large withdrawals when reviewing the books, and we alerted our bank right away. It was a substantial amount for us—we are a small business—and yet
Casting a Vote
“Every time you spend money, you’re casting a vote for the kind of world you want.” Environmentalist Anna Lappe It’s voting season here in the US, and this quote above comes to mind, but not about our politicians or economy. My friend and colleague Usha, from India, from time to time alerts me to people
Medicare… Me?
Yup. This week I turn 65, and I am officially on Medicare! It makes turning 65 a joyous event. Really! Switching healthcare policies over to Medicare was surprisingly easy. But partly this is due to my previous work on hemophilia and healthcare. Back in 2005, we were the ones to alert the community to the
