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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    BMRN A BioMarin Pharmaceutical Inc. -0.44
    ABEO A Abeona Therapeutics Inc. -5.04
    PULM A Pulmatrix, Inc. -6.22
    KRYS A Krystal Biotech, Inc. -3.34
    DSGN A Design Therapeutics, Inc. -5.08
    XFOR B X4 Pharmaceuticals, Inc. 0.42
    NGNE C Neurogene, Inc. -6.12
    TSHA C Taysha Gene Therapies, Inc. -1.63
    SWTX C SpringWorks Therapeutics, Inc. -3.72
    SNPX C Synaptogenix, Inc. -2.92
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Biotechnology Medicine Biopharmaceutical Cancer Gene Therapy Molecular Biology Autoimmune Disease Biology Genetic Engineering Life Sciences Medical Genetics Organ Systems Pompe Disease Virotherapy Achondroplasia Acquired Hemolytic Anemia Adeno Associated Virus Anemia Applied Genetics Autoimmune And Inflammatory Disease

    Related ETFs - A few ETFs which own one or more of the above listed Rare Diseases stocks.

    Symbol Grade Name Weight
    HELX C Franklin Genomic Advancements ETF 6.64
    PBE D PowerShares Dynamic Biotech & Genome 4.93
    PPH C Market Vectors Pharmaceutical ETF 4.39
    XBI D SPDR S&P Biotech ETF 3.99
    ARKG F ARK Genomic Revolution Multi-Sector ETF 3.92
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        Rare Diseases Stocks Recent News

        Date Stock Title
        Apr 18 BMRN Earnings Preview: Bristol Myers Squibb (BMY) Q1 Earnings Expected to Decline
        Apr 18 SWTX SpringWorks Therapeutics to Report First Quarter 2024 Financial Results Thursday, May 2, 2024
        Apr 17 BMRN BioMarin Pharmaceutical (BMRN) Expected to Beat Earnings Estimates: Should You Buy?
        Apr 17 KRYS Krystal Biotech (KRYS) is on the Move, Here's Why the Trend Could be Sustainable
        Apr 15 XFOR X4 Pharmaceuticals: PDUFA Excitement Ahead
        Apr 15 KRYS Recent 3.8% pullback isn't enough to hurt long-term Krystal Biotech (NASDAQ:KRYS) shareholders, they're still up 437% over 5 years
        Apr 15 TSHA Certain Biotech Investors Get an Early Look at Results. Is That Fair?
        Apr 12 BMRN Insider Sell: Biomarin Pharmaceutical Inc (BMRN) Chief Accounting Officer Erin Burkhart ...
        Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
        Related Stock Lists: Biotechnology Genodermatoses Medicine Biopharmaceutical Cancer Clinical Medicine Dermatology Disorders Epidermolysis Bullosa Gene Therapy Inflammation Lipid Lipid Storage Disorders Myotonic Dystrophy Neurological Disorders Ocular Disease Organ Systems Orphan Drug Acid Acromegaly
        Rare Diseases

        Rare diseases are medical conditions that affect a small percentage of the population. They are often genetic, chronic, and progressive, and can cause significant physical and mental disability. There are over 7,000 rare diseases, and many of them have no known cure.

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