Highlights of this month’s newsletter include:

• Gene Therapy Research, Alessandra Mortellaro, Ph.D.
• Hematopoietic Stem Cell Transplant, Hasan K. Hashem, M.D.

Highlights of this month’s newsletter include:

• Consensus Committee gathers to discuss the critical milestone of writing a consensus statement
• Family Spotlight: Anna Maria Beretta

Highlights of this month’s newsletter include:

• Patient & Family Gathering Planned
• Clinician Spotlight: Dr. Ombrello
• Family Spotlight: The Puzio Family
• Nashville, TN joins other US cities to light up monuments for Rare Disease Day

Highlights of this month’s newsletter include:

• Rare Disease Day 2021
• Researcher Spotlight: Dr. Pui Lee
• Patient Spotlight: Ishaan Jeloka
• Dr. Dan Kastner (who discovered DADA2) receives prestigious Crafoord Prize from the Royal Swedish Academy of Sciences