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    Rare Diseases Stocks List

    Symbol Grade Name % Change
    NGNE F Neurogene, Inc. 1.70
    GSK F GlaxoSmithKline PLC 0.00
    CNTG F Centogene N.V. 0.00
    AZTR F Azitra Inc. 0.00
    BMRN F BioMarin Pharmaceutical Inc. 1.42
    IONS F Ionis Pharmaceuticals, Inc. -4.12
    RLYB F Rallybio Corporaton -0.55
    BOLD F Boundless Bio, Inc. -5.17
    BLUE F bluebird bio, Inc. -1.68
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    Related Industries: Biotechnology Diagnostics & Research Drug Manufacturers - Major Drug Manufacturers - Specialty & Generic Medical Devices
    Related Stock Lists: Biotechnology Biopharmaceutical Life Sciences Anemia Cancer Drug Discovery Gene Therapy Hematology Medicine Molecule Product Organ Systems Pharmacy Syndromes Acid Adeno Associated Virus Arcturus Therapeutics Ataxia Autoimmune Disease Biologics Biology

    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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    Compare IDNA to GNOM Compare PINK to GNOM Compare PINK to IDNA

        Rare Diseases Stocks Recent News

        Date Stock Title
        Nov 21 BMRN Why Is BioMarin Pharmaceutical Inc. (BMRN) Among the Worst Performing Biotech Stocks in 2024?
        Nov 21 IONS Why Is Ionis Pharmaceuticals, Inc. (IONS) Among the Worst Performing Biotech Stocks in 2024?
        Nov 21 BMRN BioMarin: Challenges Mount, But Sale Rumors Make For A Risky 'Buy' Call
        Nov 21 BMRN Here's Why BioMarin Pharmaceutical (BMRN) is a Strong Value Stock
        Nov 20 GSK CDC warns of an imminent spike in COVID, flu cases
        Nov 20 IONS Ionis Pharmaceuticals, Inc. (IONS): Among the Best Genomics Stocks to Buy Right Now
        Nov 19 BMRN BioMarin Pharmaceutical Inc. (BMRN): Among the Best Genomics Stocks to Buy Right Now
        Nov 19 GSK GSK's Investigational Liver Disease Candidate Hits Primary Goal In Late-Stage Study To Treat Relentless Itch In Some Patients
        Nov 19 GSK GSK's Investigational Liver Disease Candidate Hits Primary Goal In Late-Stage Study To Treat Relentless Itch In Some Patients
        Nov 19 GSK GSK reports positive Phase 3 results for linerixibat in PBC itching
        Nov 19 NGNE Neurogene GAAP EPS of -$1.19 in-line
        Nov 18 NGNE Neurogene Reports Third Quarter 2024 Financial Results and Highlights Recent Updates
        Nov 18 NGNE How A Side Effect Has Wiped Out 73% Of Neurogene's Value In A Week
        Nov 18 NGNE Neurogene stock plunges 36% on NGN-401 study update
        Nov 18 IONS Here's Why Ionis Pharmaceuticals (IONS) is Poised for a Turnaround After Losing -13.46% in 4 Weeks
        Nov 18 GSK Medicus Pharma Ltd. Appoints Faisal Mehmud, MD, MRCP as Chief Medical Officer
        Nov 18 NGNE Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
        Nov 16 BMRN BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of VOXZOGO® (vosoritide) in Children with Achondroplasia at the European Society for Paediatric Endocrinology (ESPE) Meeting 2024
        Nov 16 BLUE bluebird bio Third Quarter 2024 Earnings: EPS Beats Expectations, Revenues Lag
        Nov 15 GSK How analysts are reacting to RFK Jr. as Trump's HHS pick
        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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