FDA Clears IND Application For NT-I7, a PML Treatment
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FDA Clears IND Application For NT-I7, a PML Treatment

The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…

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CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism
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CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism

  In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…

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Familial Chylomicronemia Syndrome Foundation Announces Support for HEART Act
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Familial Chylomicronemia Syndrome Foundation Announces Support for HEART Act

  In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…

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Oral Octreotide Approved to Treat Acromegaly
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Oral Octreotide Approved to Treat Acromegaly

As shared in Medscape, the FDA recently approved oral octreotide (Mycapssa) for the treatment of patients with acromegaly. These delayed-release capsules are the first approved oral somatostatin analog ever approved.…

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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines
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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines

  Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…

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Nucala Granted Priority Review For Treatment of Hypereosinophilic Syndrome
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Nucala Granted Priority Review For Treatment of Hypereosinophilic Syndrome

  According to a drug filing and press release from Pharmaceutical Business Review, the FDA granted a priority review to GlaxoSmithKline for their humanized monoclonal antibody therapy, Nucala (mepolizumab). Currently,…

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The FDA is Developing, Approving, and Expediting Exciting New Therapies
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The FDA is Developing, Approving, and Expediting Exciting New Therapies

  Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…

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Press Release: Rare Disease Community Calls on Congress & FDA to Enact Life-Saving Public Policy Solutions

Contact: Britta Vander Linden bvanderlinden@everylifefoundation.org 917.604.6518 30 million Americans need more treatment and diagnostic opportunities https://everylifefoundation.org/rare-disease-community-calls-on-congress-fda-to-enact-life-saving-public-policy-solutions/ (Washington, D.C., December 4, 2019) Hundreds of rare disease advocates from around the country were brought to Washington,…

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