At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
In a joint statement, biopharmaceutical companies AstraZeneca and Merck & Co., Inc. ("Merck") shared that LYNPARZA (olaparib) achieved a superiority boundary in the Phase 3 OlympiA clinical trial. During…
Supporting the development of my children's talents has returned the joy of life to our whole family - in spite of HAE diagnosis and other obstacles in life. As you…
Like many other residents of West Hollywood, Mark Chaney uses public transportation, specifically the bus. Twice a day, Monday through Friday, Mark boards the bus at his local stop. He…
As reported in NewsWise, as rare diseases get their time under the microscope, growing evidence uncovers medical distinctions. New lines are drawn, delineating new disorders to be defined and classified.…
Monty Hui, an eight-year-old boy from Australia, and his family have spoken to SBS News about living with a rare disease and the obstacles faced in diagnosis and recognition. He…
A recent study has illuminated the need for further research into how different forms of exercise may provide certain benefits for patients living with Charcot-Marie-Tooth disease (CMT). This study was…
There's positive news for Evrysdi, a treatment for spinal muscular atrophy (SMA). The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved, and…
Sarcoma Sarcoma is a cancer that accounts for 1% of all adult cancers. It can affect individuals of any age and can impact any part of the body. It grows…
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