Latest Breaking News On - Sedaghatian type spondylometaphyseal dysplasia - Page 1 : comparemela.com

Apr 26, 2021 5:00 AM PT Some 400 million patients worldwide are affected by more than 7,000 rare diseases; yet treatments for rare genetic diseases remain an underserved area. More than 95 percent of rare diseases do not have an approved treatment, and new treatments are estimated to cost more than $1 billion. Sanath Ramesh created the RareCamp project and the OpenTreatments Foundation to enable patients to create gene therapies for rare genetic diseases and then work with their doctors and nonprofit organizations to develop drugs. The Linux Foundation, the nonprofit organization enabling mass innovation through open source, is helping those efforts succeed. Ramesh is the father of one such patient. His two-and-one-half-year-old son was born with a rare disease, Sedaghatian-type Spondylometaphyseal Dysplasia (SSMD), which is caused by a mutation in the GPX4 gene.

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