The FDA Has Granted Orphan Drug Status to an Experimental Drug to Treat Amyloidosis

The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…

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For the First Time in 30 Years: A New Advancement in Rhabdomyosarcoma Treatment
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For the First Time in 30 Years: A New Advancement in Rhabdomyosarcoma Treatment

  According to MedPage Today, a phase III study showed that 6 additional months of chemotherapy for maintenance led to a sizable increase in the 5-year overall survival rate for…

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First Major Treatment Advance for PV Has Been Approved by the FDA
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First Major Treatment Advance for PV Has Been Approved by the FDA

  According to BioSpace, a new treatment for pemphigus vulgaris (PV) has been recently approved by the U.S. Food and Drug Administration. The treatment, Rituxan (rituximab), is geared towards adults who have…

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Orphan Drug Designation Has Been Awarded to Selumetinib for the Treatment of Neurofibromatosis Type 1
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Orphan Drug Designation Has Been Awarded to Selumetinib for the Treatment of Neurofibromatosis Type 1

The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…

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Drug Label Expanded in the UK for Treating Rare Inflammatory Diseases
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Drug Label Expanded in the UK for Treating Rare Inflammatory Diseases

According to a story from Hospital Healthcare Europe, The European Medicines Agency approved Kineret last year, an anti-inflammatory drug developed by Sobi. In the UK, Kineret is an approved treatment…

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