Catalyst Pharmaceuticals Announces Sub-Licensee DyDo Pharma Launched FIRDAPSE® in Japan
January 21, 2025 08:19 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates
January 10, 2025 08:00 ET
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Inozyme Pharma Inc.
Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates
World Orphan Drug Alliance expands to Canada and full LATAM region, completing the first truly global commercialization offering
January 10, 2025 08:00 ET
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World Orphan Drug Alliance (WODA)
World Orphan Drug Alliance expands to Canada and full LATAM region, completing the first truly global commercialization offering.
AscellaHealth Releases Q4 2024 Specialty & Rare Pipeline Digest™ Featuring Key Specialty Drug, Biosimilar, Cell and Gene Therapy Approvals
January 09, 2025 10:00 ET
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AscellaHealth, LLC
AscellaHealth Releases Q4 2024 Specialty & Rare Pipeline Digest™ Featuring Key Specialty Drug, Biosimilar, Cell and Gene Therapy Approvals
Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA
December 30, 2024 00:45 ET
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Novartis Pharma AG
Ad hoc announcement pursuant to Art. 53 LR The Phase III STEER study met its primary endpoint showing an increase from baseline in HFMSE total score in patients with SMA...
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
December 17, 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
December 16, 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
December 15, 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
December 11, 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
December 03, 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.