Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
September 24, 2024 07:30 ET
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Wave Life Sciences USA, Inc.
Mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, with high consistency across participants, in a prespecified analysis; dystrophin was comprised of two...
Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy
August 12, 2024 08:30 ET
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Wave Life Sciences USA, Inc.
Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531...
Catalyst Pharmaceuticals Enters Into an Exclusive License, Supply and Commercialization Agreement with Kye Pharmaceuticals for AGAMREE® in Canada
July 24, 2024 08:05 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., July 24, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
Muscular Dystrophy Association Applauds Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above
June 20, 2024 18:42 ET
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Muscular Dystrophy Association
New York, June 20, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the U.S. Food and Drug Administration’s expanded approval of ELEVIDYS (delandistrogene...
Catalyst Pharmaceuticals to Participate at the BofA Securities Health Care Conference 2024
May 01, 2024 08:00 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., May 01, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
Catalyst Pharmaceuticals to Participate in the 2024 Cantor Virtual Muscular Dystrophy Symposium
March 27, 2024 08:03 ET
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Catalyst Pharmaceuticals, Inc.
CORAL GABLES, Fla., March 27, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing,...
Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy
March 21, 2024 19:45 ET
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Muscular Dystrophy Association
New York, March 21, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association’s funding of foundational research leads to new drug approval by the US Food and Drug Administration (FDA) of Duvyzat...
MetrioPharm stellt wissenschaftlichen Beirat aus erfahrenen Immunologie- und Pädiatrie-Experten vor
March 13, 2024 06:00 ET
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AKAMPION
Die neuen Beiräte werden ihr Fachwissen in den Bereichen Immunologie, Pathologie, Entzündungsprozesse und in MetrioPharms Hauptindikation Duchenne-Muskeldystrophie einbringenDie Beiräte haben bereits...
MetrioPharm Establishes Scientific Advisory Board of Seasoned Immunology and Pediatric Experts
March 13, 2024 06:00 ET
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AKAMPION
The new advisors will supply expertise in immunology, pathology, inflammatory processes and in MetrioPharm's lead indication Duchenne muscular dystrophy Advisors have previously collaborated with...
Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy
February 27, 2024 08:30 ET
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Wave Life Sciences USA, Inc.
Presentations include data for WVE-N531 that provide first clinical evidence of a potential therapeutic for DMD accessing satellite cells, which are important for potential muscle regeneration ...