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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    ZVRA A Zevra Therapeutics, Inc. 1.78
    QNCX B Quince Therapeutics, Inc. 13.04
    INM B InMed Pharmaceuticals Inc. 9.93
    PULM B Pulmatrix, Inc. 8.72
    RCEL B Avita Medical Limited -1.38
    WVE C WAVE Life Sciences Ltd. 0.63
    KRYS C Krystal Biotech, Inc. 2.35
    DSGN C Design Therapeutics, Inc. 1.64
    SWTX C SpringWorks Therapeutics, Inc. -3.04
    PHVS C Pharvaris N.V. 2.63
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Epidermolysis Bullosa Genodermatoses Disease Medicine Biopharmaceutical Biotechnology Clinical Development Clinical Medicine Dermatology Life Sciences Medical Genetics Organ Systems Acid Acute Pain Aesthetics Amphetamine Angioedema Ataxia Attention Deficit Hyperactivity Disorder Bone

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

    Symbol Grade Name Weight
    PINK D Simplify Health Care ETF 10.02
    IDNA D iShares Genomics Immunology and Healthcare ETF 3.64
    GNOM F Global X Genomics & Biotechnology ETF 3.55
    BKIV A BNY Mellon Innovators ETF 3.06
    FBT D First Trust Amex Biotech Index Fund 2.96
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        Rare Diseases Stocks Recent News

        Date Stock Title
        Nov 21 ZVRA Zevra Niemann-Pick disease type C drug Miplyffa now available
        Nov 21 ZVRA Zevra Therapeutics Announces U.S. Commercial Availability of MIPLYFFA™ (arimoclomol) for Treatment of Niemann-Pick Disease Type C
        Nov 20 SWTX SpringWorks started at outperform by Evercore ahead of FDA decision
        Nov 16 ZVRA Analysts Have Made A Financial Statement On Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) Third-Quarter Report
        Nov 15 SWTX Analyst Estimates: Here's What Brokers Think Of SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) After Its Third-Quarter Report
        Nov 15 WVE Wave Life Sciences to Present at Jefferies London Healthcare Conference
        Nov 15 WVE Wave Life Sciences Ltd. (NASDAQ:WVE) Released Earnings Last Week And Analysts Lifted Their Price Target To US$22.20
        Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
        Related Stock Lists: Biopharmaceutical Biotechnology Lipid Storage Disorders Biology Medicine Alzheimer's Disease Applied Genetics Clinical Research Gene Therapy Life Sciences Organ Systems Acquired Hemolytic Anemia Amyotrophic Lateral Sclerosis Antibodies Antibody Antisense RNA Ataxia Autoimmune And Inflammatory Disease Autoimmunity C5a
        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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