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Obtaining adequate drug exposure in the brain is key to treating CNS diseases effectively. Prodrugs Remain in “Proof-of-Concept” Stages Prodrug strategies for CNS deliver have also been applied with varying success , with the most famous example being L-Dopa for Parkinson’s disease. Figure 18.
Gelbman most recently served as the founding CEO of FDNA, a digital health startup that develops advanced AI tools to help diagnose patients with rare diseases. Patel currently serves as the director of Infectious Diseases Research Laboratory at the Mayo Clinic, as well as co-director of the clinical Bacteriology Laboratory.
Next month, families affected by the rare genetic disease metachromatic leukodystrophy (MLD) will meet in Washington. Because Calliope Joy didn’t have an older sibling with the disease, her initial symptoms were confusing. It is jargony enough to reawaken nightmares of taking organicchemistry.
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