It is international Pompe Day! “Together we are Strong” Today is meant to help create awareness for the Lysosomal Storage Disorder Pompe Disease, which is both rare and underdiagnosed. Pompe…
Patients with sickle cell disease often face stigma about their need for pain medication because care providers are suspicious of drug abuse, reports The Dallas Post Tribune. Although this is…
Researchers at Northern Arizona University (NAU) are using microbial sequencing to better understand the causes of airway inflammatory diseases, including chronic rhinosinutsitis and cystic fibrosis. They hope that by studying…
Researchers at Northwestern University (NU) have published research showing that changes in the brain causing spinocerebellar ataxia type 1 (SCA1) occur at a younger age than previously thought, reports NeuroscienceNews.…
According to an announcement from the pharmaceutical company Retrophin, Inc., the company is starting to move forward with phase 3 trials for its drug candidate sparsentan. The drug is in…
La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
According to a story from medicalxpress.com, a recent study suggests that cognitive deficits that can occur as side effects of targeted brain cancer drugs could be successfully reversed with therapy.…
According to a story from the National Institutes of Health, the innovative new CRISPR gene editing technology is being used in order to identify specific genes that could play a…
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