Fetal alcohol spectrum disorders (FASDs) occur following alcohol exposure during a mother’s pregnancy. But what is life like for those with fetal alcohol syndrome and their caregivers? The Irish Examiner…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…
In a recent article for Hemophilia News Today, Jennifer Lynne, who is diagnosed with both von Willebrand disease and hemophilia B, stresses the importance of remembering women can have hemophilia…
Currently, there are a number of treatment options for patients with follicular lymphoma. But for patients whose condition has relapsed despite prior treatments, there are very limited therapeutic options. After…
Jaguar Health and Napo EU S.p.A have just announced that crofelemer, an investigative therapy for short bowel syndrome (SBS) has been granted Orphan Drug Designation by the EMA. Thanks to…
Diagnosing a rare disease early is important, as it allows for prompt intervention and treatment. Recently, researchers took large steps forward in the diagnostics of pulmonary arterial hypertension (PAH) in…
Rare disease patients know what it's like to go without a diagnosis despite enduring symptoms. Doctors often don't recognize the disorders for a number of reasons, whether that's the similarity…
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