The dance of two-steps-forward one-step-back continues.
Several treatments near FDA approval while another, for myotubular myopathy, has claimed the lives of four young children in a clinical trial and the brain degeneration of adrenoleukodystrophy (ALD, of Lorenzo’s Oil fame), continues in three of four boys given gene therapy in 2009. Earlier intervention and/or a different delivery system may be necessary; timing and dose are critical.
Still, gene therapy efforts are alive and well. On October 21, 2021, the FDA, NIH, 10 pharmaceutical companies and 5 non-profits announced joining forces “to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”