Grace Frank worked as a copy editor, city editor, reporter and news designer for leading American newspapers, including The New York Times and The International Herald Tribune, for many years. She has won numerous journalism awards, and was nominated for a Pulitzer Prize for an investigative series into eye surgeries wrongly conducted outside a clinical trial.
The U.S. Food and Drug Administration (FDA) accepted Genentech‘s application requesting approval for risdiplam, its potential oral treatment for all types of spinal muscular atrophy (SMA), and gave it priority ... Read more
AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation issues” that led to inaccuracies in its application to the ... Read more
Screening newborns for genetic diseases for which treatments exist that can prevent crippling or deadly progression, especially for rare disorders like spinal muscular atrophy, has a way to go ... Read more
Within a lifetime, spinal muscular atrophy has leapt from a disease “nobody knew anything about” to a treatable — albeit chronic and still rare — condition. It is an ... Read more
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