Global Rare Disease Therapeutics Market Outlook, 2028: Hematologic Diseases Propel Market Growth with Novel Treatments, While Injectables Take the Lead in Managing Rare Diseases
January 25, 2024 11:39 ET
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Research and Markets
Dublin, Jan. 25, 2024 (GLOBE NEWSWIRE) -- The "Rare Disease Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028" report has been added to ...
Alkeus Pharmaceuticals Appoints Seemi Khan, M.D., Chief Medical Officer
January 23, 2024 08:30 ET
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Alkeus Pharmaceuticals
Alkeus Pharmaceuticals, Inc. announces that Seemi Khan, M.D., M.P.H., M.B.A., has joined the Company as Chief Medical Officer.
Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™
January 23, 2024 01:00 ET
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Azafaros BV
Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™ Four posters with baseline data from the natural history (PRONTO) study in...
NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services
January 22, 2024 07:00 ET
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NMD Pharma
NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services Aarhus, Denmark, 22 January 2024 – NMD Pharma A/S, a clinical-stage biotech dedicated to...
Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease
January 18, 2024 03:00 ET
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Orphalan
Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease Paris, France 18 January, 2024 - Orphalan SA (“Orphalan” or “the Company”),...
Pleco Therapeutics receives FDA Orphan Drug Designation
January 16, 2024 11:30 ET
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Pleco Therapeutics B.V.
The U.S Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics BV’s lead compound, PTX-252 for the treatment of AML.
USAN Approves Generic Name “Ruxoprubart” for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases
January 08, 2024 08:30 ET
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NovelMed Therapeutics Inc
The USAN Council has officially adopted "Ruxoprubart (Ruk”soe proo' bart)" as the generic name for NM8074, marking a significant milestone in the drug development process.Ruxoprubart is an...
Zealand Pharma to participate in the Goldman Sachs Healthcare C-Suite Unscripted Conference on January 4th
January 02, 2024 02:00 ET
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Zealand Pharma
Press Release – No. 1 / 2024 Zealand Pharma to participate in the Goldman Sachs Healthcare C-Suite Unscripted Conference on January 4th Copenhagen, Denmark, January 2, 2024 – Zealand...
The Foundation for Sarcoidosis Research (FSR) awards $100,000 in support of cardiac sarcoidosis
December 18, 2023 15:41 ET
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Foundation for Sarcoidosis Research
The Foundation for Sarcoidosis Research (FSR) awards $100,000 in support of cardiac sarcoidosis.
Travere Therapeutics Initiates Pivotal Phase 3 Clinical Trial of Pegtibatinase for the Treatment of Classical Homocystinuria (HCU)
December 14, 2023 07:30 ET
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Travere Therapeutics, Inc.
SAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3...