Physicians at the Canadian Specialist Hospital in Dubai operated on a one-day-old baby with a birth defect called spina bifida, says a recent report. The surgery saved the infant’s life.…
According to an article from FinancialBuzz, the pharmaceutical company Avadel announced that its product FT 218 has been granted orphan drug status by the Food and Drug Administration (FDA) as…
A new fundraising challenge is sweeping all social media platforms once again for amyotrophic lateral sclerosis (ALS), first reported Mom.me. Back in 2014, the ice bucket challenge was a wild success, being…
As an adult living with rare disease, there have been significant gaps in my employment history. I am sure many adults with health complications experience their workforce years this way,…
Researchers from St. Jude Children's Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID,…
London specialists at the King's College hospital believe they should take an 11-month-old baby Isaiah suffering from multiple diseases off life support to avoid any further pain, reported The Guardian.…
Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…
The Australian government has announced that almost that new treatments worth $300 million are being added to the Pharmaceutical Benefits Scheme. This program provides subsidized medical treatments to citizens of…
According to an article from the Chicago Tribune, many medical specialists are coming to the realization that the eligibility criteria for clinical trials should be reassessed. The problem is that…
Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…
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