26 Patient Volunteer Leaders Attend the Foundation for Sarcoidosis Research Global Sarcoidosis Clinic Alliance Volunteer Patient Leadership Advanced Training Conference held at Cleveland Clinic
March 27, 2024 06:41 ET
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Foundation for Sarcoidosis Research
26 Patient Volunteer Leaders joined the Foundation for Sarcoidosis Research at Cleveland Clinic for the Leadership Advanced Training Conference
Inozyme Pharma to Report Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency and ENPP1 Deficiency on April 8, 2024
March 26, 2024 08:30 ET
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Inozyme Pharma Inc.
Inozyme announced that it will host a conference call and webcast on Monday, Apr. 8, 2024 at 8:00 a.m. ET.
NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US
March 22, 2024 07:00 ET
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NMD Pharma
NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US Aarhus, Denmark, 22 March 2024 – NMD Pharma A/S, a...
NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US
March 22, 2024 02:00 ET
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NMD Pharma
NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US Aarhus, Denmark, 22 March 2024 – NMD Pharma A/S, a...
CAMP4 Therapeutics Announces Dosing of First Participant in Phase 1 Clinical Study of CMP-CPS-001, a Potential First-in-Class Therapeutic for Urea Cycle Disorders
March 21, 2024 07:58 ET
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CAMP4 Therapeutics
CAMP4 Therapeutics Announces Dosing of First Participant in Phase 1 Clinical Study of CMP-CPS-001, a Potential First-in-Class Therapeutic for Urea Cycle D
NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine
March 21, 2024 07:00 ET
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NMD Pharma
NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens...
NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine
March 21, 2024 02:00 ET
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NMD Pharma
NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens...
North America Rare Disease Therapeutics Market Set for Significant Growth, with Projected 10.88% CAGR Through 2028
March 20, 2024 13:30 ET
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Research and Markets
Dublin, March 20, 2024 (GLOBE NEWSWIRE) -- The "North America Rare Disease Therapeutics Market, Competition, Forecast & Opportunities, 2018-2028" report has been added to ...
Quoin Pharmaceuticals Provides Corporate Update and Announces Fourth Quarter and 2023 Financial Results
March 13, 2024 16:05 ET
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Quoin Pharmaceuticals, Inc.
Company reported positive initial clinical data for QRX003 from first six evaluable subjects in ongoing open-label Netherton Syndrome study with positive benefits observed across a number of clinical...
Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne Muscular Dystrophy (DMD)
March 13, 2024 07:55 ET
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Catalyst Pharmaceuticals, Inc.
AGAMREE® (vamorolone) a Novel Alternative Corticosteroid with Demonstrated Properties in Maintaining Efficacy and a Well-Tolerated Side Effect Profile Available in the U.S. by Prescription for...