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.

    New Results Published from Phase 3 Clinical Trial Evaluating Berotralstat for HAE

      On October 22, 2020, BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from a Phase 3 clinical trial evaluating berotralstat for patients with hereditary angioedema (HAE). As a…

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    Dravet Syndrome Treatment FINTEPLA (Fenfluramine) Receives Positive CHMP Opinion
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    Dravet Syndrome Treatment FINTEPLA (Fenfluramine) Receives Positive CHMP Opinion

      In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While…

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    New Research Finds 28 Genes Associated with Developmental Disorders, Bringing Diagnoses to Families
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    New Research Finds 28 Genes Associated with Developmental Disorders, Bringing Diagnoses to Families

    At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…

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    Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure
    Quinton's Quest for a Cure (photo submitted by Quinton's mother, Laura Nitahara)

    Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure

    When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…

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    Fibryga Now Indicated to Treat Factor I Deficiency
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    Fibryga Now Indicated to Treat Factor I Deficiency

      According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by…

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    Rett Syndrome Treatment TSHA-102 Received Rare Pediatric Disease and Orphan Drug Designations

    Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…

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    UT Southwestern Expands Diagnostic and Treatment Approaches for Aplastic Anemia, MDS
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    UT Southwestern Expands Diagnostic and Treatment Approaches for Aplastic Anemia, MDS

    What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…

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    IB1001 Effective for NPC Treatment, Study Says
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    IB1001 Effective for NPC Treatment, Study Says

    According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…

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    New Collaboration: Scribe Therapeutics and Biogen to Explore CRISPR-Related Therapies for ALS

    On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…

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    5-Year STELARA Data Shows Impact on Patients with Crohn’s Disease
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    5-Year STELARA Data Shows Impact on Patients with Crohn’s Disease

      Recently, during the virtual 28th United European Gastroenterology Week Congress, the Janssen Pharmaceutical Companies of Johnson & Johnson announced 5-year results from a Phase 3 extension study on STELARA…

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    One Month Ago, HealthWell Foundation Opened the Adrenal Insufficiency Fund
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    One Month Ago, HealthWell Foundation Opened the Adrenal Insufficiency Fund

      About one month ago, the HealthWell Foundation announced the beginning of the Adrenal Insufficiency Fund. Designed to assist patients with adrenal insufficiency, the fund will grant financial assistance to…

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    Phase 3 SIDEROS Trial for DMD Drug Idebenone Fails
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    Phase 3 SIDEROS Trial for DMD Drug Idebenone Fails

    While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…

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    First Patient with Alzheimer’s Dosed in Bryostatin-1 Clinical Trial
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    First Patient with Alzheimer’s Dosed in Bryostatin-1 Clinical Trial

      Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1…

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    Scientists Discovered the First Effective Alkaptonuria Treatment

    In the past, there existed no approved treatments for alkaptonuria (AKU), a rare genetic disorder characterized by homogentisate dioxygenase deficiency. However, according to Medical XPress, this recently changed as British…

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    A Simple Sugar Could Be the Key to Treating Multiple Sclerosis
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    A Simple Sugar Could Be the Key to Treating Multiple Sclerosis

    In conditions like multiple sclerosis or neuromyelitis optica, disease progression is spurred by the destruction of myelin, the protective coating around nerve cells. However, new research suggests that N-acetylglucosamine, a…

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