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
|
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
|
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
|
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
|
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
|
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
|
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
|
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
|
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...
Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled
December 11, 2023 01:00 ET
|
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...
Catalyst Pharmaceuticals Receives Prestigious Ranking on Forbes 2024 List of America's Most Successful Small-Cap Companies
December 05, 2023 08:03 ET
|
Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., Dec. 05, 2023 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX) today announced its inclusion in Forbes' esteemed list of America's Most...