Jim and Jill Kelly founded Hunter’s Hope in 1997 after their son, Hunter, was diagnosed with Krabbe when he was just a few months old. Most children who have this disease do not live past their ...

The deal grants GEMMABio exclusive, worldwide rights to develop and commercialize PBGM01 for GM1 gangliosidosis, PBKR03 for ...

There are two types of lipoid pneumonia: Endogenous lipoid pneumonia results from systemic conditions, such as Krabbe and Niemann-Pick disease, in which lipids accumulate within the lung.

They realized their granddaughter Makayla had died exactly five years earlier. She had Krabbe disease—a rare neurodegenerative illness that strikes infants and usually kills them in less than ...