Catalyst Pharmaceuticals will present real-world findings on Duchenne muscular dystrophy at the 2026 MDA Conference in Orlando.
Quiver AI Summary
Catalyst Pharmaceuticals, Inc. announced its participation in the 2026 MDA Clinical & Scientific Conference in Orlando, Florida, where it will present real-world findings on Duchenne muscular dystrophy (DMD) through multiple poster presentations. The company will also sponsor a vamorolone-focused Industry Forum alongside Santhera Pharmaceuticals. Chief Medical Officer Will Andrews emphasized the importance of sharing insights that enhance patient care within the DMD community, highlighting the role of AGAMREE® in treatment. Presentations will include analyses of cardiac medication use with vamorolone and the impact of glucocorticoid treatment on healthcare resource utilization and respiratory decline in DMD patients. Catalyst continues to focus on improving treatments for rare diseases and ensuring patient access to necessary care.
Potential Positives
- Catalyst Pharmaceuticals will showcase real-world findings in Duchenne muscular dystrophy (DMD) at a major industry conference, which can enhance its visibility and reputation in the biopharmaceutical field.
- The company is contributing to advancements in DMD treatment through rigorous research, potentially positioning itself as a leader in this therapeutic area.
- The recognition of Catalyst as one of America’s Most Successful Companies by Forbes and inclusion in the Deloitte Technology Fast 500™ list highlights its growth and success, attracting potential investors and partners.
Potential Negatives
- The press release highlights the company's participation in an event focused on Duchenne muscular dystrophy, which may suggest the company's primary focus and market is niche, potentially limiting broader market appeal.
- The mention of known and unknown risks in the forward-looking statements could indicate underlying uncertainties about the company's future performance and strategies.
- The press release does not provide specific new data or results regarding their products, which may lead to questions about the progress and efficacy of their treatments.
FAQ
What is the focus of Catalyst Pharmaceuticals?
Catalyst Pharmaceuticals specializes in developing and commercializing novel medicines for rare and difficult-to-treat diseases.
Where will Catalyst present its findings on Duchenne muscular dystrophy?
Catalyst will present at the 2026 MDA Clinical & Scientific Conference in Orlando, Florida.
What is vamorolone?
Vamorolone is a novel corticosteroid being studied by Catalyst Pharmaceuticals for its potential in treating Duchenne muscular dystrophy.
Who are the featured speakers at the MDA Industry Forum?
Speakers include Dr. Craig McDonald and Dr. Leanne Ward, experts in pediatric rehabilitation and physical medicine.
What recognition has Catalyst Pharmaceuticals received recently?
Catalyst has been named one of America's Most Successful Companies by Forbes for three consecutive years (2023-2025).
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
CORAL GABLES, Fla., March 06, 2026 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today announced that the Company will showcase real‑world findings in Duchenne muscular dystrophy (DMD) through multiple poster presentations at the 2026 MDA Clinical & Scientific Conference in Orlando, Florida. Catalyst will also sponsor a vamorolone-focused MDA Industry Forum with Santhera Pharmaceuticals.
“The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our poster presentations and our sponsored symposium on long‑term evidence generation, we’re contributing clinical insights that help clarify AGAMREE ® ’s profile and its role in patient care. Our commitment is to advance rigorous, meaningful research that moves the field forward for patients and their families.”
The poster presentations will focus on real-world analysis of vamorolone in patients living with DMD:
Poster Presentations:
Abstract Title:
Real-world analysis of concomitant cardiac medication use with the novel corticosteroid vamorolone in patients with Duchenne muscular dystrophy (DMD)
Authors:
Linda Cripe, MD, Nationwide Children's Hospital, Steven Woods, PharmD, Catalyst, Katherine Habkouk, PharmD, AnovoRx Specialty Pharmacy, Regina Grebla, PhD, Northeast Epi, LLC
Session Name:
Poster Session
Topic:
Clinical Management
Poster Number:
74 S
Date of Presentation:
Sunday, March 8, 2026, 6:00 PM–8:00 PM ET
Abstract Title:
Association Between Glucocorticoid Treatment Duration and Health Care Resource Utilization in Duchenne Muscular Dystrophy: A Real-World Analysis.
Authors:
Steven Woods, PharmD, Catalyst Pharmaceuticals, Bridget McGowan, MD, Ann and Robert H. Lurie Children's Hospital of Chicago, Ashley Martin, PhD, BluePath Solutions, Alexa Gordon, MS, BluePath Solutions, Sana Mirza, MPH, BluePath Solutions, Paula Alvarez, MBA, MS, Rph, Critical Intelligence Consulting
Session Name:
Poster Session
Topic:
Other
Poster Number:
75 S
Date of Presentation:
Sunday, March 8, 2026, 6:00 PM–8:00 PM ET
Abstract Title:
Glucocorticoid Use and Delayed Respiratory Decline in Duchenne Muscular Dystrophy Patients: A Real-World Analysis
Authors:
Steven Woods, PharmD, Catalyst Pharmaceuticals, Bridget McGowan, MD, Ann and Robert H. Lurie Children's Hospital of Chicago, Ashley Martin, PhD, BluePath Solutions, Alexa Gordon, MS, BluePath Solutions, Sana Mirza, MPH, BluePath Solutions, Paula Alvarez, MBA, MS, Rph, Critical Intelligence Consulting, Oscar Mayer, MD, The Children's Hospital of Philadelphia
Session Name:
Poster Session
Topic:
Other
Poster Number:
76S
Date of Presentation:
Sunday, March 8, 2026, 6:00 PM–8:00 PM ET
MDA Industry Forum will highlight the latest insights from clinical leaders advancing our understanding of DMD:
Symposium :
Title:
MDA Industry Forum Sponsored by Catalyst Pharmaceuticals in Conjunction With Santhera Pharmaceuticals
Speakers:
- Dr. Craig McDonald, MD, Chair, Department of Physical Medicine & Rehabilitation Professor, Departments of Pediatrics and Physical Medicine & Rehabilitation UC Davis Health, Sacramento, CA
- Dr. Leanne Ward, MD, Professor of Pediatrics, University of Ottawa, Ottawa, ON
Date of Presentation:
Tuesday, March 10, 2026, 12:00 PM-1:30 PM ET
Location:
Key Largo Room, The Hilton Orlando
About Catalyst Pharmaceuticals, Inc.
Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence, which remains the cornerstone of our commercial strategy, while continuously evaluating strategic opportunities to expand our global footprint. Catalyst, headquartered in Coral Gables, Fla., has been recognized by Forbes as one of America’s Most Successful Company in 2023, 2024, and 2025, and on the 2025 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies.
For more information, please visit Catalyst's website at www.catalystpharma.com .
Forward-Looking Statements
This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors described in Catalyst's Annual Report on Form 10-K for the 2025 fiscal year and its subsequent filings with the U.S. Securities and Exchange Commission (“SEC”), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
Contact Information:
Investor Contact
Melissa Kendis, Catalyst Pharmaceuticals, Inc.
(305) 420-3200
[email protected]
Media Contact
Ignacio Guerrero-Ros, Ph.D., Russo Partners, LLC
(646) 249-6817
[email protected]
