The modern rare disease market has evolved dramatically over the past decade. Success is no longer measured solely by seizure reduction. Investors, physicians, caregivers, regulators, and payers increasingly expect therapies that improve neurological development, functional outcomes, quality of life, and caregiver burden. Diseases such as CDKL5 Deficiency Disorder, Rett syndrome, Fragile X syndrome, Angelman syndrome, and Dravet syndrome represent some of the most scientifically challenging—and commercially attractive—areas in biotechnology. Commercializing…
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