According to a story from SFGate, many survivors of childhood cancers are plagued with financial burdens for the rest of their lives. This is because treatment of childhood cancer can…
The US FDA has awarded Orphan Drug status to the investigational drug NSI-189 for use by people with Angelman syndrome. You can read the source article here, at Nasdaq. About…
According to a story from The Guardian, a recent study suggests that cannabidiol, or CBD, could magnify the effectiveness of treatment for pancreatic cancer. CBD is naturally found in the…
The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…
According to MedPage Today, a phase III study showed that 6 additional months of chemotherapy for maintenance led to a sizable increase in the 5-year overall survival rate for…
According to News Medical, a team of researchers from London have created new technologies that may aid in further understanding Duchenne muscular dystrophy (DMD). Not only this, but the new…
According to BioSpace, a new treatment for pemphigus vulgaris (PV) has been recently approved by the U.S. Food and Drug Administration. The treatment, Rituxan (rituximab), is geared towards adults who have…
The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…
According to a story from Hospital Healthcare Europe, The European Medicines Agency approved Kineret last year, an anti-inflammatory drug developed by Sobi. In the UK, Kineret is an approved treatment…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
