New Research for Idiopathic Pulmonary Fibrosis to Investigate Genomic Cause of Disease

Idiopathic pulmonary fibrosis (IPF) is a rare condition with no known cause and no effective treatments besides a lung transplant. Current therapies focus on slowing progression of the disease but…

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New Potential Treatment for Neurofibromatosis Type 1 Given Breakthrough Therapy Designation

A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…

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Researchers are Working Hard to Understand GNAO1 Mutations: A Condition so Rare it Doesn’t Yet have a Name

"When only 100 people in the world share your child’s specific gene mutation, it is hard to find anyone who understands, including doctors." Rare diseases are at a disadvantage when…

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Gene Therapy for Beta Thalassemia Recommended for EU Approval

According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has…

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Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…

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