Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates
10 janv. 2025 08h00 HE
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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
10 janv. 2025 08h00 HE
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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
09 janv. 2025 10h00 HE
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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
30 déc. 2024 00h45 HE
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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
17 déc. 2024 06h00 HE
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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 déc. 2024 08h03 HE
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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 déc. 2024 13h45 HE
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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 déc. 2024 01h00 HE
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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 déc. 2024 07h00 HE
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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 nov. 2024 13h51 HE
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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