Targeted Delivery: BPM31510 is a CoQ10-lipid nanoparticle conjugate designed for intravenous administration to deliver ...
Designation highlights significant unmet medical needAUSTIN, Texas, Jan. 20, 2026 (GLOBE NEWSWIRE) -- Rein Therapeutics ...
Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...
Japan's Ministry of Health, Labour and Welfare (MHLW) grants MCO-010 Sakigake (Pioneering Regenerative Medical Product) and ...
The FDA's orphan drug designation supports LP-284's development for soft tissue sarcomas, addressing significant unmet needs in this adult patient population. The FDA granted orphan drug designation ...
"Cellenkos gains FDA orphan drug status for CK0804 to treat myelofibrosis" was originally created and published by ...
Sixth overall for Lantern’s A.I.-driven precision oncology pipelineOrphan designation expands LP-284's potential for an ...
Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...
The FDA designates ARB1002 as an orphan drug, highlighting its potential as a novel treatment for pancreatic cancer amid ...
Rising concerns about spending on prescription drugs that treat rare diseases are not justified, according to a new analysis in the journal Health Affairs. "We wanted to focus on the true impact of ...
The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug exclusion, a once narrow exception that permitted manufacturers of rare disease ...
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