Happy Friday Everyone! As we get ready for the weekend, we want to spotlight four articles. We have an article about a man with Parkinson's who uses social media to…
According to a story from Business Wire, the therapeutics company Alnylam Pharmaceuticals recently announced that the company's drug patisiran has gained approval from the European Commission. Patisiran was approved for…
Cynata Therapeutics, an Australian stem cell and regenerative medicine company, has announced positive results from a Phase 1 clinical trial of CYP-001, the CymerusTM mesenchymal stem cell product candidate. The study…
According to a story from statnews.com, Dr. Julia Brody, who heads the Silent Spring Institute located in Newton, Massachusetts, says that her laboratory has always allowed patients and study participants…
According to a story from the Santa Cruz Sentinel, the earliest results from a critical gene editing study could offer some encouragement for those hoping to treat rare genetic diseases…
The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…
Based on a report by Parkinson’s News Today, a recent study confirmed the safety of an increasingly popular Parkinson’s treatment. The treatment, known as deep brain stimulation, has long been…
The US Food and Drug Administration has approved a drug called TakhzyroTM (lanadelumab-flyo) for the treatment of hereditary angioedema. This approval was based on supporting evidence from several studies, including from…
Another article previously published under "Featured Adult: Rebekah Palmer's Story" in The Cystinosis Advocate/ Volume 10, Issue 1/ Spring and Summer 2017: I feel an obstacle in being the adult patient…
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