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Alkeus Pharmaceuticals Appoints Seemi Khan, M.D., Chief Medical Officer
23 janv. 2024 08h30 HE | 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.
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Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™
23 janv. 2024 01h00 HE | 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...
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NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services
22 janv. 2024 07h00 HE | 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...
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Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease
18 janv. 2024 03h00 HE | 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”),...
Ivo Timmermans
Pleco Therapeutics receives FDA Orphan Drug Designation
16 janv. 2024 11h30 HE | 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.
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USAN Approves Generic Name “Ruxoprubart” for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases
08 janv. 2024 08h30 HE | 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...
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Zealand Pharma to participate in the Goldman Sachs Healthcare C-Suite Unscripted Conference on January 4th
02 janv. 2024 02h00 HE | 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...
cardiac grant awardees
The Foundation for Sarcoidosis Research (FSR) awards $100,000 in support of cardiac sarcoidosis
18 déc. 2023 15h41 HE | Foundation for Sarcoidosis Research
The Foundation for Sarcoidosis Research (FSR) awards $100,000 in support of cardiac sarcoidosis.
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Travere Therapeutics Initiates Pivotal Phase 3 Clinical Trial of Pegtibatinase for the Treatment of Classical Homocystinuria (HCU)
14 déc. 2023 07h30 HE | 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...
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Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled
11 déc. 2023 01h00 HE | Azafaros BV
          Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled Topline data from the study, expected for mid-2024, will support two pivotal Phase...