10:27:50 PM PDT - Monday, August 2nd, 2021

Dying Patients With Rare Diseases Struggle to Get Experimental Therapies  

By Editor - Wed Jul 14, 2:48 am

  • Comments Off on Dying Patients With Rare Diseases Struggle to Get Experimental Therapies

At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntington’s disease. “There’s lots of things that she used to be able to do just fine,” said her mom, Londen Tabor, who lives with her daughter in Gillette, Wyoming. Autumn’s speech has become slurred and her cognitive skills slower. She needs help with many tasks, such as writing, showering and dressing, and while she can walk, her balance is off. Autumn has been turned down for clinical trials because she is too young. “It is so frustrating to me,” Tabor said. “I would sell my soul to try to get any type [of treatment] to help my daughter.” For patients like Autumn with serious or immediately life-threatening conditions who do not qualify for clinical trials and have exhausted all treatment options, there may be another option: seeking approval from the Food and Drug Administration for expanded access , or compassionate use, of experimental therapies.

More:

Dying Patients With Rare Diseases Struggle to Get Experimental Therapies

  • Comments Off on Dying Patients With Rare Diseases Struggle to Get Experimental Therapies

Leave a Reply

Comments are closed on this post.