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News

Explore the latest stories and publications from NCATS and our partners, see upcoming events, or explore the variety of resources we provide to better understand translational science and our work.

News

Read the latest news from NCATS and its collaborators whose research is supported through the Center’s programs, or follow coverage of NCATS' translational research activities through national and local media.

Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy

May 15, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

World’s First Patient Treated With Personalized CRISPR Gene Editing Therapy at Children’s Hospital of Philadelphia

May 15, 2025 - Grantee/Partner News

  • Our Impact on Rare Diseases
  • Stem Cell Translation Laboratory (SCTL)

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment

May 15, 2025 - Grantee/Partner News

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

How Do Middle-Aged Folks Get Dementia? It Could Be These Proteins

May 14, 2025 - Grantee/Partner News

  • Our Impact on Clinical Trials

You and Your Pet Can Share Resistance to Certain Medications. Here’s How.

May 13, 2025 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

Protein in the Blood Can Help Predict ALS Survival and Decline, Play Role in Developing Treatments

May 13, 2025 - NCATS News

  • Our Impact on Clinical Trials
  • Our Impact on Rare Diseases
  • Rare Diseases Clinical Research Network (RDCRN)

Researchers showed how the level of a protein found in the blood might be used in developing new drugs to treat ALS.

Last updated on September 29, 2026