Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

    ICYMI: FDA Clears CNTY-101 IND Application for Lupus
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    ICYMI: FDA Clears CNTY-101 IND Application for Lupus

    A drug cannot be transported or distributed across state lines until its marketing application has been approved/cleared, so getting this clearance is crucial for many drug developers and physicians who…

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    ICYMI: Kanuma for Lysosomal Acid Lipase Deficiency Now Approved in England
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    ICYMI: Kanuma for Lysosomal Acid Lipase Deficiency Now Approved in England

    In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…

    Continue Reading ICYMI: Kanuma for Lysosomal Acid Lipase Deficiency Now Approved in England
    Researchers Identify Potential CDKL5 Treatment Target
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    Researchers Identify Potential CDKL5 Treatment Target

      As it stands today, there are no cures for CDKL5 deficiency disorder. Treatment is symptomatic and supportive, and includes physical, occupational, and speech therapy. CDKL5 deficiency disorder causes seizures,…

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    A Phase 1/2 Trial on AIV001 for Basal Cell Carcinoma is Now Complete
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    A Phase 1/2 Trial on AIV001 for Basal Cell Carcinoma is Now Complete

      AIV001 (axitinib): a potential, non-surgical therapeutic option for people with basal cell carcinoma (BCC). AiViva Biopharma describes AIV001 as: a novel formulation of a multi-kinase inhibitor combined with AiViva’s…

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    Oral Arsenic Trioxide Regimen Effective for Treating APL, Study Results Suggests

      The 66th American Society for Hematology (ASH) Annual Meeting and Exhibition took place from December 7-10, 2023. During the meeting, researchers and other stakeholders shared insights into clinical trends,…

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    ICYMI: TAR-200 for High-Risk Non-Muscle-Invasive Bladder Cancer Granted Breakthrough Therapy Designation
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    ICYMI: TAR-200 for High-Risk Non-Muscle-Invasive Bladder Cancer Granted Breakthrough Therapy Designation

    In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that…

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    Vyjuvek for DEB Earns Orphan Drug Designation in Japan
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    Vyjuvek for DEB Earns Orphan Drug Designation in Japan

    Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…

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    STUDY: Keytruda + mRNA-4157 (V940) Vaccine Reduced Risk of Severe Melanoma Recurrence
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    STUDY: Keytruda + mRNA-4157 (V940) Vaccine Reduced Risk of Severe Melanoma Recurrence

    In an investor news release from December 2023, Moderna and Merck shared follow-up data from the Phase 2b KEYNOTE-492/mRNA-4157-P201 clinical trial. During the trial, researchers explored the impact of Keytruda…

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    ICYMI: LP-284 Earns Orphan Drug Designation for High-Grade B-Cell Lymphoma with MYC/BCL2 Rearrangements
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    ICYMI: LP-284 Earns Orphan Drug Designation for High-Grade B-Cell Lymphoma with MYC/BCL2 Rearrangements

      Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat,…

    Continue Reading ICYMI: LP-284 Earns Orphan Drug Designation for High-Grade B-Cell Lymphoma with MYC/BCL2 Rearrangements
    Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study
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    Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study

      Last year, the FDA placed a clinical hold on an Investigational New Drug (IND) application for deucrictibant for the on-demand and prophylactic treatment of individuals living with hereditary angioedema…

    Continue Reading Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study
    ICYMI: Keytruda and Chemo Combination Approved for Locally Advanced Unresectable or Metastatic Biliary Tract Cancer
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    ICYMI: Keytruda and Chemo Combination Approved for Locally Advanced Unresectable or Metastatic Biliary Tract Cancer

      Also known as cholangiocarcinoma, biliary tract cancer can be incredibly aggressive. Further, this cancer is often diagnosed in later stages, making it more difficult to treat. The 5-year survival rate currently…

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    The NFL’s My Cause My Cleats 2023: Lacing Up for Awareness with Players’ Personal Stories 
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    The NFL’s My Cause My Cleats 2023: Lacing Up for Awareness with Players’ Personal Stories 

      During the NFL’s My Cause My Cleats initiative, passion and purpose collide on the field. This initiative embodies the profound bond between athletes and the causes that matter to…

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