It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
As she stood in the garden, surrounded by friends and family, a splitting headache richocheted through Ciara Wilkie's head. Her vision blurred. At first, Ciara didn't think much of it.…
When it comes to treating interstitial cystitis, doctors may take a multipronged approach: physical therapy, bladder distention, surgery, nerve stimulation, oral medications. But people with this condition lack therapies…
The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
As recently reported by Forbes, the U.S. Food and Drug Administration (FDA) approved Leqembi (lecanemab) for Alzheimer’s disease earlier this week. Leqembi is a humanized monoclonal antibody that targets…
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