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This research service provides an overview of the orphan drugs market dynamics, estimating the market size broken down by different segments. It also dives deeper into the different technological, geographical, and therapeutic opportunities with an objective assessment of the disclosed pipelines. Notably, the study has an expanded coverage of not only rare diseases, but also hard-to-cure and commercially non-viable diseases.
Rare diseases have been an increasing area of focus as three waves have converged in recent years: genomic research and innovations becoming mainstream; the forthcoming regulatory policies and financial incentives put in place by the federal agencies; and the increasingly patient-centric and coordinated efforts of patient advocacy groups, caregivers, and centers of excellence (COEs).
New Therapeutic Avenue for Rare Pediatric Diseases by Angela Mohan on February 10, 2021 at 2:19 PM
Scientists have devised a new approach for detecting and potentially heading off the effects of two rare pediatric diseases named Beckwith-Wiedemann syndrome and Silver-Russell syndrome before birth, as per the study performed in mouse models of the diseases and published in
Cell Reports.
Both diseases result in growth-related symptoms in children and often lead to additional problems later in life, such as increased cancer risk from Beckwith-Wiedemann syndrome and increased metabolic disease risk from Silver-Russell syndrome. Both of these diseases have lifelong consequences, said Piroska Szabó, Ph.D., an associate professor at Van Andel Institute and the study s corresponding author. Our findings provide a critical foundation for additional studies that we hope will translate into new, life-changing prenatal detection and treatment methods. Our goal is