According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
According to a story from The Jerusalem Post, Adrian Krainer is a prominent molecular geneticist and biochemist. His research was also pivotal in developing the first-ever disease modifying treatment for…
According to a publication from Fabry Disease News, the U.S. Food and Drug Administration (FDA) has approved American biotechnology company Avrobio's Investigational New Drug (IND) application for experimental Fabry disease…
Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…
May is Cystic Fibrosis (CF) awareness month! CF is one of the more devastating rare diseases in the community, often affecting children and young people. So let's be extra mindful…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…
The Origins of Fighting H.A.R.D. The Fighting H.A.R.D. Foundation was established in 2015 by two sisters who both live with multiple rare diagnoses. 16-year-old Allie is diagnosed with juvenile rheumatoid…
Google Backs Startup For Treating Heart Disease Via CRISPR Injections May 16, 2019 --Sharing is Caring-- Google Backs Startup For Treating Heart Disease Via CRISPR Injections Once in our life…
According to a story from BioSpace, the drug developer Biogen recently released interim results from a Phase 1/2 clinical trial. This clinical trial is testing the drug tofersen, which is…
This week, a press release from the office of South Carolina senator Lindsey Graham announced the designation of May 17th as Glioblastoma Awareness Day. The Senate Resolution was cosponsored by…
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