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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    BMRN A BioMarin Pharmaceutical Inc. -10.91
    XFOR B X4 Pharmaceuticals, Inc. -5.43
    BOLD B Boundless Bio, Inc. -1.81
    DSGN B Design Therapeutics, Inc. -0.82
    KRYS B Krystal Biotech, Inc. -2.02
    PULM B Pulmatrix, Inc. 3.96
    TSHA C Taysha Gene Therapies, Inc. -6.38
    GSK C GlaxoSmithKline PLC 0.15
    SWTX C SpringWorks Therapeutics, Inc. 2.17
    SRPT C Sarepta Therapeutics, Inc. 1.63
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Biotechnology Biopharmaceutical Cancer Life Sciences Clinical Development Gene Therapy Medicine Duchenne Muscular Dystrophy Genetic Diseases Infectious Diseases Inflammation Influenza Muscular Dystrophy Myotonic Dystrophy Pompe Disease Achondroplasia Acid Antacid Antibiotics Antisense RNA

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

    Symbol Grade Name Weight
    LABD B Direxion Daily S&P Biotech Bear 3X Shares 1.87
    IBRN D iShares Neuroscience and Healthcare ETF 1.06
    BIS B ProShares UltraShort Nasdaq Biotechnology 0.65
    BBP D BioShares Biotechnology Products Fund 0.62
    MOON F Direxion Moonshot Innovators ETF 0.53
    View all Rare Diseases ETFs...
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    Compare BIS to MOON Compare LABD to BIS Compare LABD to IBRN

        Rare Diseases Stocks Recent News

        Date Stock Title
        Apr 25 SRPT Bullish Sarepta Therapeutics Insiders Loaded Up On US$13.3m Of Stock
        Apr 25 BMRN Q1 2024 Biomarin Pharmaceutical Inc Earnings Call
        Apr 25 BMRN Biomarin Pharmaceutical Inc (BMRN) Q1 2024 Earnings Call Transcript Highlights: Strong Growth ...
        Apr 25 BMRN BioMarin Pharmaceutical, Inc. (BMRN) Q1 2024 Earnings Call Transcript
        Apr 25 BMRN BioMarin Pharmaceutical Inc. 2024 Q1 - Results - Earnings Call Presentation
        Apr 24 BMRN BioMarin (BMRN) Q1 Earnings: Taking a Look at Key Metrics Versus Estimates
        Apr 24 BMRN BioMarin Pharmaceutical Inc. (BMRN) Q1 2024 Earnings: Surpasses Revenue and EPS Estimates
        Apr 24 BMRN BioMarin updated 2024 guidance sees EPS raise, but no change to revenue
        Apr 24 BMRN BioMarin Pharmaceutical (BMRN) Tops Q1 Earnings Estimates
        Apr 24 BMRN BioMarin Pharmaceutical Non-GAAP EPS of $0.71 beats by $0.11, revenue of $648.83M misses by $2.98M
        Apr 24 BMRN BioMarin Reports Record Financial Results for the First Quarter 2024
        Apr 24 SWTX SpringWorks Therapeutics Announces Abstracts Accepted for Presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting
        Apr 24 GSK GSK (GSK) Earnings Expected to Grow: What to Know Ahead of Next Week's Release
        Apr 24 SRPT Sarepta Therapeutics to Announce First Quarter 2024 Financial Results
        Apr 23 BMRN BioMarin Pharmaceutical Q1 2024 Earnings Preview
        Apr 23 BMRN Is a Surprise Coming for BioMarin (BMRN) This Earnings Season?
        Apr 23 BMRN AstraZeneca (AZN) to Report Q1 Earnings: Here's What to Expect
        Apr 23 KRYS Down -7.21% in 4 Weeks, Here's Why Krystal Biotech (KRYS) Looks Ripe for a Turnaround
        Apr 22 BMRN BioMarin Pharmaceutical Investors to Focus Q1 on Strategic Review, Cost-Cutting Measures, UBS Says
        Apr 22 GSK Sanofi reportedly settles 4K Zantac cases for $100M
        Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
        Related Stock Lists: Biotechnology Gene Therapy Biology Emerging Technologies Life Sciences Lipid Medicine Metabolic Disorders Molecular Biology Severe And Rare Diseases Acute Pain Adeno Associated Virus Aesthetics Anemia Antibodies Antibody Applied Genetics Biomarkers Biopharmaceutical Blood Diseases
        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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