What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.
Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook…
Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.”
Spinal muscular atrophy…Continue
Like any of you who participated in today’s Advisory Committee Meeting at the FDA for PTC Therapeutic’s ataluren, I am emotionally exhausted. Whether you participated by speaking during the Open Public Hearing, attending the Ad Comm on the FDA campus, submitting your written testimony, or…Continue