The ULF is happy to share this press release on behalf of Forge Biologics regarding their clinical trial for patients with Krabbe. Here is a preview but click the link below to read the full press release.
UPDATE: As of Tuesday, January 30: The ACHDNC has recommended the addition of Krabbe disease to the RUSP!
“We continue to be encouraged by the safety and efficacy observed in FBX-101 treated patients,” stated Dr. Escolar. “The window of treatment for patients with Krabbe disease is very narrow and newborn screening is a critical step for these babies to benefit from any treatment, but unfortunately newborn screening for this devastating and quickly progressing disease is still only available in a handful of states. Dr. Miller’s comments to the ACHDNC will reflect the urgency physicians have been trying to convey for this patient community.”