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December 18, 2025 14:20 ET | Source: Foundation for Sarcoidosis Research

Foundation for Sarcoidosis Research Publishes Comprehensive Voice of the Patient Report Following Externally Led PFDD Meeting with the FDA

The FSR Voice of the Patient report outlines a clear path forward for drug developers, researchers, clinicians, and regulators for sarcoidosis.
December 15, 2025 08:03 ET | Source: Catalyst Pharmaceuticals, Inc.

Catalyst Pharmaceuticals to Participate in the 44th Annual J.P. Morgan Healthcare Conference

CORAL GABLES, Fla., Dec. 15, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
December 11, 2025 08:00 ET | Source: Hope for PDCD Foundation

Hope for PDCD Foundation Delivers Petition to FDA Urging Approval of the First Targeted PDCD Treatment

Hope for PDCD Foundation submitted a petition to FDA urging approval of the first targeted potential treatment for PDCD.
December 10, 2025 09:20 ET | Source: Ocugen

Ocugen CEO to Present at Oppenheimer Movers in Rare Disease Summit

MALVERN, Pa., Dec. 10, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that...
December 09, 2025 07:30 ET | Source: Novartis Pharma AG

Novartis ianalumab significantly extends disease control in patients with immune thrombocytopenia with only four once-monthly doses

Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45% with patients maintaining disease control 2.8 times longer than with placebo plus eltrombopag1,2 62% of patients treated with...
December 08, 2025 09:00 ET | Source: Muscular Dystrophy Association

John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), to Deliver Keynote Address on the Future of Biotech and Rare Disease Innovation at the 2026 MDA Clinical & Scientific Conference

New York, Dec. 08, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) announced today that John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), will...
December 08, 2025 08:00 ET | Source: Soleo Health Holdings, Inc.

Soleo Health Appoints Jody Thompson as New Senior Vice President of Business Development, Rare Disease

Soleo Health appoints Jody Thompson as SVP of Business Development to expand portfolio of exclusive and limited distribution therapies for rare diseases.
December 08, 2025 02:00 ET | Source: Pharming Group N.V.

Pharming Group to participate in Oppenheimer Movers in Rare Disease Summit

Pharming to participate in Oppenheimer Movers in Rare Disease Summit on Dec 11 in NYC. Chief Medical Officer Anurag Relan in fireside chat at 1:50 PM ET.
November 25, 2025 07:00 ET | Source: Purespring Therapeutics

Purespring Therapeutics to Present at Piper Sandler’s 37th Annual Healthcare Conference

Purespring Therapeutics to Present at Piper Sandler’s 37th Annual Healthcare Conference London – 25 November 2025 - Purespring Therapeutics, a precision nephrology company leading the development...
November 24, 2025 08:03 ET | Source: Catalyst Pharmaceuticals, Inc.

Catalyst Pharmaceuticals to Participate in the Bank of America CNS Therapeutics Virtual Conference

CORAL GABLES, Fla., Nov. 24, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...