The Health Resources and Services Administration (HRSA) has announced the decision to add metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) to the federal Recommended Uniform Screening Panel (RUSP). IGT Chairman Congressman Erik Paulsen issued the following statement:   “IGT is grateful for the leadership of U.S. Department of Health and Human Services Secretary Robert F. Kennedy, Jr. and HRSA Administrator Tom Engels in recognizing the urgent need for

For Dr. Allyson Berent, science and motherhood collided the day her daughter was diagnosed with Angelman syndrome. Today, as Chief Science Officer at the Foundation for Angelman Syndrome Therapeutics (FAST), she’s turning that urgency into progress, advancing the first gene therapy programs for Angelman syndrome and reshaping how rare disease research moves from discovery to treatment. She also co-founded AS 2 Bio, a drug development accelerator that is building a new model f

Kendra Riley is a public relations professional, mother, and a strong advocate for gene therapies. In 2020, two of her daughters were...

Nate Plasman is a Duchenne Muscular Dystrophy (DMD) parent advocate, a devoted husband to his wife of 20 years, Sara, and a proud father...

Lauren Holder is a research ambassador, advocate, and committed caregiver. After discovering she was gene-positive for Huntington’s...

On June 17, the Institute for Gene Therapies (IGT), in partnership with the Alliance for Regenerative Medicine (ARM), convened a panel...

Amanda Moore is a devoted mother, advocate, and CEO of the Angelman Syndrome Foundation. After adopting her two sons, Jackson and Baden,...

Lesa Brackbill is a passionate mother, advocate, author, and member of IGT’s Patient Advocacy Advisory Council. After her daughter...

Authors: IGT Chairman Erik Paulsen, Scientific Academic and Medical Council Chair Donna Christian-Christensen, M.D. As our nation’s...

As we commemorate the fifth anniversary of the Institute for Gene Therapies (IGT) today, I want to take a look back at the strides we've...