Patient Worthy was pleased to serve on the advisory committee for the development of this new resource. Caring for a child with a rare disease can be scary and confusing;…
The National Organization for Rare Disorders (NORD) has been a leading patient advocacy organization since the 1980s, helping advance the identification, treatment, and cure of rare disorders. In October 2021,…
Ashley Monroe is an extremely talented singer-songwriter who hails from Tennessee. Over the years, Ashley has grown her music career alone and as part of the Pistol Annies. But now…
When it comes to pharmaceuticals, licensed territory helps ensure that drugs within a certain territory are offered to patients. Exclusive licenses sometimes mean that pharmaceuticals are only available in specific…
For rare diseases in which no current treatment options are available, such as GM1 gangliosidosis, there is an urgent need to find therapeutics and improve patient outcomes. Gene therapy company…
Meghan Thompson, a doctor and 3rd year fellow at Memorial Sloan Kettering Cancer Center has recently explained all of the emerging treatments using BTK inhibitors to treat CLL, chronic lymphocytic…
Paroxysmal nocturnal hemoglobinuria (PNH) occurs from a mutated PIG-Agene. This causes blood cells to be produced which don't have surface proteins. As such, the cells aren't protected adequately from the complement…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
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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.
