EyeDNA Therapeutics Receives Rare Pediatric Disease Designation from FDA for its Investigational Gene Therapy HORA-PDE6b for Patients with Retinal Dystrophy due to PDE6b Gene Mutations
17. Dezember 2024 06:00 ET
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Coave Therapeutics
PDE6b-related Retinitis Pigmentosa is a rare inherited retinal dystrophy affecting up to 3,000 people in the US Symptoms often start in childhood leading to blindness by midlife with no approved...
Catalyst Pharmaceuticals to Present at the 43rd Annual J.P. Morgan Healthcare Conference
16. Dezember 2024 08:03 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., Dec. 16, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
Pharming announces public cash offer to the shareholders of Abliva AB
15. Dezember 2024 13:45 ET
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Pharming Group N.V.
Proposed acquisition strengthens Pharming’s late-stage pipeline with a potential first-in-disease asset Abliva’s lead product KL1333 is currently in a pivotal clinical trial, with a positive interim...
Pharming announces positive topline data in pediatric clinical trial of leniolisib
11. Dezember 2024 01:00 ET
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Pharming Group N.V.
Multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency Data consistent with the improvements seen in the previously...
Chemomab to Participate in Oppenheimer’s Movers in Rare Disease Summit
03. Dezember 2024 07:00 ET
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Chemomab Therapeutics
Chemomab management is participating in the Oppenheimer Movers in Rare Disease Summit on Dec 13, 2024 at the Westin Grand Central in NYC.
FSR Doubles Investment in Cardiac Sarcoidosis Research with $200,000 in Grants to Advance Diagnostic and Treatment Breakthroughs
27. November 2024 13:51 ET
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Foundation for Sarcoidosis Research
FSR Doubles Investment in Cardiac Sarcoidosis Research with $200,000 in Grants to Advance Diagnostic and Treatment Breakthroughs
Catalyst Pharmaceuticals Recognized as One of North America’s Fastest-Growing Companies on the 2024 Deloitte Technology Fast 500™ List
25. November 2024 08:03 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., Nov. 25, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), today announced it ranked number 452 on the Deloitte Technology Fast...
Catalyst Pharmaceuticals to Participate in Upcoming Investor Conferences
19. November 2024 08:03 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., Nov. 19, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
Alkeus Pharmaceuticals Receives FDA Rare Pediatric Disease and Fast Track Designations for Gildeuretinol as a Treatment for Stargardt Disease
18. November 2024 09:16 ET
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Alkeus Pharmaceuticals
Alkeus Pharmaceuticals, Inc. receives FDA Rare Pediatric Disease and Fast Track designations for gildeuretinol as a treatment for Stargardt Disease
Fortress Biotech Reports Third Quarter 2024 Financial Results and Recent Corporate Highlights
14. November 2024 16:10 ET
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Fortress Biotech, Inc.
Emrosi approved by FDA on November 4 for the treatment of inflammatory lesions of rosacea in adults Cosibelimab PDUFA goal date of December 28 for potential approval to treat metastatic or locally...