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Top News In Orphanet Journal Today - Breaking & Trending Today

Duchenne Muscular Dystrophy Global Market Report 2023

Major players in the duchenne muscular dystrophy market are Bristol Myers Squibb Company, FibroGen Inc., Italfarmaco SpA, NS Pharma Inc., Pfizer Inc., PTC. ....

New York , United States , United Kingdom , South Korea , Eli Lilly , University Of Texas Southwestern , Akashi Therapeutics Inc , Aavantibio Inc , Capricor Therapeutics Inc , Nippon Shinyaku Co Ltd , Hamon Center , Acceleron Pharma Inc , Biomarin Pharmaceutical Inc , Muscular Dystrophy Global Market Report , Acceleron Pharma , Capricor Therapeutics , Western Europe , Eastern Europe , North America , South America , Middle East , Orphanet Journal , Rare Diseases , Biomed Central , United Kingdom Based , Texas Southwestern ,

Rhythm Pharmaceuticals (RYTM) Announces Two Publications Detailing Burden of Hyperphagia and Obesity on Patients and Caregivers Living with Bardet-Biedl Syndrome

Rhythm Pharmaceuticals (RYTM) Announces Two Publications Detailing Burden of Hyperphagia and Obesity on Patients and Caregivers Living with Bardet-Biedl Syndrome
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United States , United Kingdom , Hannah Deresiewicz , David Connolly , Andream Haqq , Adam Daley , David Meeker , Stern Investor Relations Inc , Corporate Communications , Rhythm Pharmaceuticals Inc , Drug Administration , United Kingdom Medicines Healthcare Products Regulatory Agency , University Of Alberta , European Commission , European Union , Department Of Pediatrics , Berry Company Public Relations , Rhythm Pharmaceuticals , Exchange Commission , Bardet Biedl Syndrome , Orphanet Journal , Multi Country Survey , Chief Executive Officer , Healthcare Products Regulatory Agency , Prescribing Information , Important Safety ,

Uncontrolled Disease Common in Patients Grappling With Myasthenia Gravis

A real-world study of 5 European countries shows frequent misdiagnoses/delays in diagnosis, acute hospitalizations, and quality-of-life impairments.
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United Kingdom , United States , European Union , Orphanet Journal ,

Assessing the Impact of the Orphan Drug Act 40 Years Later

The act was passed in 1983 and has contributed to increased research and development on treatments for rare diseases.
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United States , Drug Designation Program , Orphanet Journal , Orphan Drug Act , Orphan Drug Designation Program , Orphan Drug Designation Program , Orphan Drug , Orphan Drug Approvals , Rare Disease Drug Development ,

Genomenon Wins Best of Show Award at Bio-IT World 2023 Conference and Expo

Ann Arbor, MI (PRWEB) May 30, 2023 Genomenon Inc., a genomic intelligence company, today announced that it was awarded Best of Show at Bio-IT World ....

United States , Inozyme Pharma , Mike Klein , Life Sciences , Genomenon Inc , Show Awards , Genomic Intelligence , Genomic Language Processing , Orphanet Journal , Rare Diseases , Rare Disease Prevalence , Press Release ,