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

I just want to help Dylan. He's my baby brother.' The baby boy is currently at a hospital in Pittsburgh after being diagnosed with Krabbe disease - a rare and often lethal neurological condition.

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 ...

It was also the fifth anniversary of the death of his granddaughter who suffered from Krabbe disease, a rare neurodegenerative illness. He and his wife allocated nearly $30 million of their ...