Through its recently issued press release, bluebird bio announced the FDA’s accelerated approval of the first therapy to slow the progression of adrenoleukodystrophy (ALD). The disease is the result…
Are you ready for the October 2022 Dysautonomia Challenge? You can make this October stand out on your calendar by making each day meaningful, event ladened, exciting, and dedicated…
On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
The young patients began laughing, and some even began to speak and walk. Imagine the excitement among the hospital staff when they realized that a new medication brought about…
Don't forget to read Part 1, where we discuss Dr. Maleddu's background, why she chose to specialize in medical oncology, what a desmoid tumor is, and its symptoms, causes, and treatments.…
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