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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 déc. 2024 06h00 HE | 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...
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Catalyst Pharmaceuticals to Present at the 43rd Annual J.P. Morgan Healthcare Conference
16 déc. 2024 08h03 HE | 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,...
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Pharming announces public cash offer to the shareholders of Abliva AB
15 déc. 2024 13h45 HE | 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...
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Pharming announces positive topline data in pediatric clinical trial of leniolisib
11 déc. 2024 01h00 HE | 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...
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Chemomab to Participate in Oppenheimer’s Movers in Rare Disease Summit
03 déc. 2024 07h00 HE | 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 Cardiac Grant Announcement (Facebook Ad)
FSR Doubles Investment in Cardiac Sarcoidosis Research with $200,000 in Grants to Advance Diagnostic and Treatment Breakthroughs
27 nov. 2024 13h51 HE | Foundation for Sarcoidosis Research
FSR Doubles Investment in Cardiac Sarcoidosis Research with $200,000 in Grants to Advance Diagnostic and Treatment Breakthroughs
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Catalyst Pharmaceuticals Recognized as One of North America’s Fastest-Growing Companies on the 2024 Deloitte Technology Fast 500™ List
25 nov. 2024 08h03 HE | 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...
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Catalyst Pharmaceuticals to Participate in Upcoming Investor Conferences
19 nov. 2024 08h03 HE | 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,...
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Alkeus Pharmaceuticals Receives FDA Rare Pediatric Disease and Fast Track Designations for Gildeuretinol as a Treatment for Stargardt Disease
18 nov. 2024 09h16 HE | Alkeus Pharmaceuticals
Alkeus Pharmaceuticals, Inc. receives FDA Rare Pediatric Disease and Fast Track designations for gildeuretinol as a treatment for Stargardt Disease
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Fortress Biotech Reports Third Quarter 2024 Financial Results and Recent Corporate Highlights
14 nov. 2024 16h10 HE | 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...