The line between genius and madness is often closer than people realize. Don’t tell Dr. Sergio Canavero that he can’t help his patient with spinal muscular atrophy (SMA). How many…
Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is complicated. Researchers have so much…
At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News Journal, his parents knew something…
The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…
In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the Charcot-Marie Tooth (CMT) community to tell…
Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after clinical trials, especially the INPULSIS…
Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…
A new public cord blood donation center recently opened up in New York. This is the second of this type of facility to open in the state. In the past,…
Adyn’s Dream is a charity in Athens, Ohio. The organization provides live events that benefit families and their children that have received a diagnosis of spinal muscular atrophy (SMA). The…
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