FDA has rejected or reversed course on five gene therapies for rare diseases while simultaneously unveiling expedited pathways for new treatment approvals.
Two years ago, Megan Selser was folding her 7-week-old infant’s clothes, clutching his fuzzy red head to her chest, when her phone rang. She answered and heard on the other end the pained voice she had used herself many times as an ICU nurse deliveri... [1186 chars]