• Mashup Score: 2
    Evaluating Nerandomilast in Patients With Rare Pulmonary Fibrotic Conditions - 20 day(s) ago

    Leticia Orsatti, MD, discusses results from clinical trials evaluating nerandomilast in patients with IPF or PPF.

    Source: CheckRare.com
    Categories: General Medicine News
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      Leticia Orsatti, MD, discusses results from clinical trials examining the safety and efficacy of nerandomilast to treat patients with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF). https://t.co/B0rlKAAWfq #CheckRare #RareLung #RareDisease https://t.co/W0yIhqsVC7

  • Mashup Score: 0
    2025 Orphan Drugs: PDUFA Dates and FDA Approvals - 21 day(s) ago

    Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2025.

    Source: CheckRare.com
    Categories: General Medicine News
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      đź“Ł Stay up to date on the most recent FDA approvals and PDUFA dates in the rare disease space with our 2025 Orphan Drugs webpage! https://t.co/zXa34ufpB8 #CheckRare #RareDiseases #PDUFADates #FDAApproval https://t.co/OCZliydrAD

  • Mashup Score: 0
    Priority Review Voucher Tracking - 22 day(s) ago

    The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases.

    Source: CheckRare.com
    Categories: General Medicine News
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      The FDA Priority Review Voucher (PRV) program aims to incentivize the development of drugs for rare pediatric and tropical diseases. Click here to visit CheckRare’s chart tracking issued PRVs and their current status https://t.co/mEpVeB6NnC #CheckRare #PriorityReview https://t.co/SGj84hz3g8

  • Mashup Score: 1
    Disease Severity and Progression in Patients With ENPP1 Deficiency - 23 day(s) ago

    Matt Winton, PhD, Inozyme Pharma, discusses an analysis characterizing disease severity and progression in patients with ENPP1 deficiency.

    Source: CheckRare.com
    Categories: General Medicine News
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      Matt Winton, PhD, Senior Vice President and Chief Operating Officer of Inozyme Pharma, discusses results from an analysis characterizing disease severity and progression in patients with ENPP1 deficiency. https://t.co/QIiaLXvWUs #CheckRare #ENPP1 #RareMetabolic #RareGenetic https://t.co/HWPnkMUpor

  • Mashup Score: 4
    Rare Disease Recognition and Awareness Days - 27 day(s) ago

    Stay informed with our comprehensive calendar of rare disease awareness days, highlighting key dates and resources.

    Source: CheckRare.com
    Categories: General Medicine News
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      NEW: Check out our Rare Disease Recognition and Awareness Days page to stay up to date on the latest rare disease awareness programs. https://t.co/TJsNXKf30j #CheckRare #RareDisease #RareDiseaseAwareness https://t.co/Hbdr2OFOrI

  • Mashup Score: 1
    A Family’s Experience With ALSP - 28 day(s) ago

    Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with ALSP.

    Source: CheckRare.com
    Categories: General Medicine News
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      Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). https://t.co/LD2dZ5eHyf #CheckRare #ALSP #RareNeurology #RareGenetic https://t.co/2bsGlvINWf

  • Mashup Score: 0
    Genetic Testing for WHIM Syndrome - 29 day(s) ago

    Jolan Walter, PhD, MD, Division Chief of Pediatric Allergy and Immunology at the University of South Florida, discusses genetic testing for WHIM syndrome.

    Source: CheckRare.com
    Categories: General Medicine News
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      Genetic Testing for WHIM Syndrome: reducing barriers, proper panel analysis, and how to deal with variants of uncertain significance. Learn more at https://t.co/PUX8CHQMx1 #CheckRare #WHIMSyndrome #RareGenetic #RareAutoimmune #RareSkin https://t.co/BUKb12GYvR

  • Mashup Score: 1
    The Current Landscape of SYNGAP1-Related Disorders - 30 day(s) ago

    Mike Graglia, Co-Founder & Managing Director of the SynGAP Research Fund, discusses the current landscape of SYNGAP1-related disorders (SRD).

    Source: CheckRare.com
    Categories: General Medicine News
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      đź’ˇRare Disease Spotlight: SYNGAP1-Related Disorders Learn more about this rare disease with our latest article https://t.co/eaHEQHJOAX #CheckRare #RareDisease #SYNGAP1 #RareNeurology #RareGenetic https://t.co/u1yyTZnmLG

  • Mashup Score: 0
    Repurposing Shelved Assets for Rare Diseases - 1 month(s) ago

    Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases.

    Source: CheckRare.com
    Categories: General Medicine News
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      Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases. https://t.co/Idi5g47b9l #CheckRare #RareDisease https://t.co/XG9Wpzgxwz

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