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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    RARE F Ultragenyx Pharmaceutical Inc. -0.82
    TAK D Takeda Pharmaceutical Company Limited 0.30
    LRMR D Larimar Therapeutics, Inc. 0.82
    ABEO D Abeona Therapeutics Inc. 0.70
    KROS D Keros Therapeutics, Inc. -0.55
    ALDX D Aldeyra Therapeutics, Inc. 1.05
    TARA D ArTara Therapeutics, Inc. -0.87
    AURA C Aura Biosciences, Inc. 3.20
    TSHA C Taysha Gene Therapies, Inc. 8.46
    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
    PPH D Market Vectors Pharmaceutical ETF 4.6
    HRTS F Tema Cardiovascular and Metabolic ETF 3.81
    GINX D SGI Enhanced Global Income ETF 3.79
    GNOM F Global X Genomics & Biotechnology ETF 3.63
    BBC F Virtus LifeSci Biotech Clinical Trials ETF 2.99
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    Compare HRTS to GINX Compare PPH to BBC Compare PPH to HRTS

        Rare Diseases Stocks Recent News

        Date Stock Title
        Nov 21 TAK Takeda and Alloy Therapeutics partner to make off-the-shelf CAR-T therapies
        Nov 20 TAK Takeda in deal with Alloy Therapeutics to develop cell therapy platforms for cancer
        Nov 19 ALDX ALDX Up as FDA Accepts Resubmitted NDA for Eye Drug Reproxalap
        Nov 19 ABEO ABEO: FDA Accepts BLA Re-Submission for Pz-cel; PDUFA Date of April 29, 2025; Raising Valuation to $9.50…
        Nov 18 ALDX Aldeyra announces FDA acceptance of resubmitted reproxalap application
        Nov 18 LRMR Larimar Therapeutics Presents Additional Data from Phase 1 Studies and Phase 2 Dose Exploration Study Supporting the Nomlabofusp Clinical Program at ICAR 2024
        Nov 18 ALDX Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease, Expands AbbVie Option Agreement
        Nov 15 TARA Protara Therapeutics to Present New Interim Data from Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC at the 25th Annual Meeting of the Society of Urologic Oncology
        Nov 15 TSHA Taysha Gene Therapies Third Quarter 2024 Earnings: Revenues Beat Expectations, EPS Lags
        Nov 15 ABEO Abeona Therapeutics Inc (ABEO) Q3 2024 Earnings Call Highlights: Strategic Advances Amid ...
        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
        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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