The events of the past week-- and the past few hundred years-- have brought up a lot of sadness, anger, and reckoning in our country. There are so many ways…
As reported in Guru Focus, biotech company Protagonist Therapeutics has announced progression in their Phase 2 study on PTG-300 as treatment for patients with polycythemia vera, a rare cancer. Dr.…
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
Being at peace with ourselves and our lives makes daily living so much easier. Of course, sometimes it's easier said than done. But one tactic may help improve comfort, confidence,…
By Danielle Bradshaw from In The Cloud Copy Researchers in Germany have gone on record saying that there is a concern with how well patients are able to tolerate the…
By Jodee Redmond from In The Cloud Copy The results of a new study using laboratory rats to model cerebral palsy with spasticity (muscle stiffness) accompanied by dystonia (involuntary muscle…
Genetic research is a burgeoning and exciting field. With the discovery of new genetic mutations and variants comes a deeper understanding of rare diseases and their subtypes. According to Cancer…
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NORD has received thousands of phone calls and emails each week from concerned rare disease patients. They are asked for advice and how to decipher the myriad of contradictory information…
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