Patients First in Rare Diseases

Dr. Maria Escolar

For rare disease families, time is the real enemy. Meet our Chief Medical Officer, Dr. Maria Escolar, and learn how a single conversation with a patient’s mother sparked a decade of research that has led to the creation of a revolutionary shift in the gene therapy approach for treating patients with Krabbe disease.

#leukodystrophyawarenessmonth #genetherapy #patientsfirst #forgebiologics

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