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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    KRYS B Krystal Biotech, Inc. 1.37
    SRPT B Sarepta Therapeutics, Inc. -3.53
    IONS B Ionis Pharmaceuticals, Inc. 5.19
    MREO C Mereo BioPharma Group plc -6.91
    LXEO C Lexeo Therapeutics, Inc. -0.76
    DNTH C Dianthus Therapeutics, Inc. -5.02
    BMRN C BioMarin Pharmaceutical Inc. -0.20
    AKTX C Akari Therapeutics Plc -24.32
    PULM C Pulmatrix, Inc. 1.04
    NGNE C Neurogene, Inc. -2.05
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Biopharmaceutical Biotechnology Clinical Development Drug Discovery Gene Therapy Medicine Organ Systems Antisense RNA Cancer Cardiomyopathy Clinical Research Dystrophy Genetic Diseases Therapeutic Gene Modulation Achondroplasia Acquired Hemolytic Anemia Acromegaly Alcohol Alport Syndrome Amyloid

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

    Symbol Grade Name Weight
    PINK A Simplify Health Care ETF 10.91
    PBE B PowerShares Dynamic Biotech & Genome 8.56
    HELX B Franklin Genomic Advancements ETF 7.93
    GNOM B Global X Genomics & Biotechnology ETF 7.21
    BBP B BioShares Biotechnology Products Fund 6.35
    View all Rare Diseases ETFs...
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        Rare Diseases Stocks Recent News

        Date Stock Title
        Jun 25 IONS Ionis gains as anti-lipid therapy undergoes FDA priority review
        Jun 25 IONS Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed
        Jun 25 IONS Ionis Pharmaceuticals (IONS) Soars 6.8%: Is Further Upside Left in the Stock?
        Jun 24 SRPT Sarepta Therapeutics Scores 90-Plus RS Rating On Muscular Dystrophy Approval
        Jun 24 SRPT Micron To Rally Around 23%? Here Are 10 Top Analyst Forecasts For Monday
        Jun 24 SRPT Company News for Jun 24, 2024
        Jun 24 SRPT Sarepta Therapeutics (SRPT) Soars 30.1%: Is Further Upside Left in the Stock?
        Jun 23 SRPT DraftKings And Dell Were Among The 10 Biggest Large Cap Stock Gainers Last Week (June 16-June 22): Are These In Your Portfolio?
        Jun 23 BMRN BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Shares Could Be 41% Below Their Intrinsic Value Estimate
        Jun 22 SRPT Trending stocks of the week: NVDA, ADSK, RKLB and more
        Jun 22 SRPT Sarepta Therapeutics: Pushing The Edge And Creating The Horizon
        Jun 21 SRPT What next for gas prices, how the S&P 500 can reach 6,000: Market Domination
        Jun 21 SRPT Growth Stocks Leave Value Stocks In The Dust: 4 Reasons For Biggest Monthly Lead In Over A Year
        Jun 21 SRPT Sector Update: Health Care Stocks Gain in Late Afternoon
        Jun 21 SRPT Sarepta Therapeutics: Why Today's Rally On DMD Drug Label Expansion Won't Last
        Jun 21 SRPT Top Stock Movers Now: FactSet, Sarepta Therapeutics, Spirit AeroSystems, and More
        Jun 21 SRPT US indexes open mixed, pharmaceutical stocks climb: Morning Brief
        Jun 21 DNTH Dianthus Therapeutics: Complement System Candidate Not Sufficiently Derisked
        Jun 21 SRPT Sarepta Stock Surges On FDA News; Industrial Stock In Buy Zone
        Jun 21 SRPT Stocks to Watch Friday: Nvidia, Micron, Sarepta, CarMax
        Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
        Related Stock Lists: Biopharmaceutical Biotechnology Clinical Development Drug Discovery Gene Therapy Medicine Organ Systems Antisense RNA Cancer Cardiomyopathy Clinical Research Dystrophy Genetic Diseases Therapeutic Gene Modulation Achondroplasia Acquired Hemolytic Anemia Acromegaly Alcohol Alport Syndrome Amyloid
        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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