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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    SWTX D SpringWorks Therapeutics, Inc. 4.06
    KROS D Keros Therapeutics, Inc. 6.08
    XFOR D X4 Pharmaceuticals, Inc. 6.48
    PASG D Passage Bio, Inc. -2.24
    NGNE D Neurogene, Inc. 3.50
    TSHA D Taysha Gene Therapies, Inc. -0.39
    LIFE D aTyr Pharma, Inc. 0.63
    AURA D Aura Biosciences, Inc. 1.54
    LRMR D Larimar Therapeutics, Inc. 2.04
    GBIO D Generation Bio Co. 1.93
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Biotechnology Life Sciences Biopharmaceutical Cancer Gene Therapy Organ Systems Central Nervous System Disorders Gene Delivery Lipid Storage Disorders Medicine Monogenic Anemia Applied Genetics Ataxia Biology Bone Bone Disease Clinical Development Clinical Pharmacology Deficiency

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

    Symbol Grade Name Weight
    IWC B iShares Microcap ETF 0.04
    IWM C iShares Russell 2000 ETF 0.01
    IWN B iShares Russell 2000 Value ETF 0.01
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        Rare Diseases Stocks Recent News

        Date Stock Title
        May 3 KROS Keros Therapeutics files automatic mixed shelf
        May 3 SWTX Here's Why We're Not Too Worried About SpringWorks Therapeutics' (NASDAQ:SWTX) Cash Burn Situation
        May 3 SWTX SpringWorks Therapeutics Inc (SWTX) (Q1 2024) Earnings Call Transcript Highlights: Navigating ...
        May 2 LIFE aTyr Pharma GAAP EPS of -$0.23 beats by $0.02, revenue of $0.24M beats by $0.22M
        May 2 LIFE aTyr Pharma Announces First Quarter 2024 Results and Provides Corporate Update
        May 2 TSHA Will Taysha Gene Therapies, Inc. (TSHA) Report Negative Q1 Earnings? What You Should Know
        May 2 TSHA Taysha Gene Therapies Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome
        May 2 SWTX SpringWorks Therapeutics GAAP EPS of -$1.18, revenue of $21M
        May 2 SWTX SpringWorks Therapeutics Reports First Quarter 2024 Financial Results and Highlights Recent Business Updates
        May 1 SWTX SpringWorks Therapeutics Q1 2024 Earnings Preview
        Apr 30 XFOR X4 Pharmaceuticals to Report First-Quarter 2024 Financial Results and Host a Conference Call and Webcast on May 7, 2024
        Apr 30 XFOR XFOR: XOLREMDI™ Approved by FDA…
        Apr 29 XFOR X4 Pharmaceuticals: FDA Approval For WHIM Warrants Further Upside
        Apr 29 TSHA These 2 ‘Strong Buy’ Penny Stocks Could Rally to $9, Says Piper Sandler
        Apr 29 XFOR X4 gets FDA approval for Xolremdi for WHIM syndrome
        Apr 29 XFOR UPDATE 1-US FDA approves X4 Pharmaceuticals' therapy for immunodeficiency disease
        Apr 29 XFOR X4 Pharmaceuticals Announces FDA Approval of XOLREMDI™ (mavorixafor) Capsules, First Drug Indicated in Patients with WHIM Syndrome
        Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
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        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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