Comment from Gayle Butterfield
AnonymousSupportIndividual
Summary: A parent of a woman with spinocerebellar Ataxia type 3 expresses support for the proposed guidance to accelerate scientific research and individualized therapies. The commenter argues that patients with rare genetic diseases need fewer regulatory hurdles and more funding to develop life-altering treatments.
My daughter has spinocerebellar Ataxia type 3 a rare disease. She was a fully functioning normal developing young woman until she started having choking and walking issues. She is only 27 years old and is struggling with walking and speech. She needs and deserves every opportunity that is scientifically possible for her to delay the devastating effects of this disease. A cure could be possible if funding and research continues. She does not need more red tape. She needs advancement of science so she can live a productive life full of all that others take for granted.