On Thursday, April 11, 2019, the Subcommittee on Labor, Health and Human Services, Education, and Related Agencies convened in Washington D.C. to review the FY2020 Budget Request for the National Institutes of Health (NIH). NIH Director, the Honorable Francis S. Collins, MD, PhD, participated in the hearing. In his witness statement, Dr. Collins references the CReATe consortium for its research in the field of ALS and related disorders." We have been heartened by the success ... of a gene therapy for the infantile version of ALS, called spinal muscular atrophy (SMA), which actually has worked quite well,” said Dr. Collins. “Now the question is, could that approach be successful, at least for some individuals with ALS where we know what the genetic mutation is? "Watch a portion of Dr. Collins’ testimony below to learn more.
Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications. Catch up on the latest RDCRN…