Scientists in NCATS’ Therapeutics for Rare and Neglected Diseases (#TRND) program helped develop the first #GeneTherapy to be delivered directly to the brain! The recently FDA-approved gene therapy, called Kebilidi, treats aromatic L-amino acid decarboxylase (AADC) deficiency –– a fatal genetic disease affecting the brain and central nervous system –– by restoring a missing AADC enzyme. Read more about this study: https://go.nih.gov/pbXSoQM #RareDisease
Caregiving is a profound act of love and commitment, but it can also be an incredibly challenging journey when you're caring for someone with a #raredisease. From navigating complex medical systems to providing round-the-clock care, #caregivers shoulder immense responsibilities that often go unnoticed by the broader public. In honor of National Family Caregivers Month, we're sharing just a few of their personal stories as a reminder of the critical role they serve in managing and caring for those with a rare condition. 📖 Read more: https://lnkd.in/erDh2rT9 #NFCM
