Rare disease drug development, which now accounts for nearly one-third of all drugs in active research & development worldwide, presents challenges that will accelerate the adoption of new development strategies…
The Leukemia Research medical journal published results from a study that showed efficacy when treating Acute Myeloid Leukemia (AML). Great news for the rare disease community! Onconova Therapeutics announced the publication of results…
Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%. This is the first FDA-approved treatment…
If you’re one of the small number of people living with acromegaly symptoms today (an estimated 60 out of every million people worldwide), we’ve got some good news for you.…
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