Browsing Tag
Duchenne muscular dystrophy
31 posts
Greenstone Biosciences wins NHLBI R61 award to identify small-molecule treatments for DMD heart disease
Greenstone Biosciences has secured a $625,968 National Heart, Lung, and Blood Institute (NHLBI) R61 grant to combine patient-derived stem cells, proteomics and artificial intelligence in the search for small-molecule treatments for cardiac fibrosis in Duchenne muscular dystrophy (DMD). The award validates the company’s human-cell-based discovery platform, but target selection, compound validation and progression into the programme’s R33 phase remain critical tests.
Deramiocel’s 9 to 3 FDA panel defeat wipes out half of Capricor’s value before August decision
Capricor loses half its value after a 9 to 3 FDA vote puts deramiocel approval, an $80 million milestone and its launch strategy at risk.
Can Secretome Therapeutics turn its expanded leadership team into a Duchenne breakthrough?
Secretome Therapeutics has recruited experienced clinical, operational, medical affairs and finance executives as the private biotechnology company prepares to move STM-01 deeper into development for Duchenne muscular dystrophy-associated cardiomyopathy. The appointments indicate that Secretome is building the organisation required for late-stage trials and potential commercialisation, although the ultimate value of that infrastructure will depend on whether STM-01 produces convincing clinical evidence.
Catalyst Pharmaceuticals (CPRX) agrees $4.1bn buyout as Angelini Pharma plans to enter US rare disease market
Angelini Pharma will pay $4.1 billion for Catalyst Pharmaceuticals at $31.50 a share, but CPRX closed at $31.10. The premium sits in the strategy, not the price.
Atossa Therapeutics (NASDAQ: ATOS) deepens Z-endoxifen bet as 2025 losses widen and rare disease optionality grows
Atossa Therapeutics widened its 2025 loss as Z-endoxifen spending rose and rare disease optionality expanded. Read what this could mean for ATOS.
A single appeals court ruling puts gene therapy patent economics back in play for Regenxbio Inc. and Sarepta Therapeutics Inc.
Regenxbio Inc. wins a key appeals court reversal against Sarepta Therapeutics Inc. Discover what this means for gene therapy IP and investors.
Will Sarepta’s Elevidys succeed where others stalled? All eyes on EMBARK trial’s 3-year data drop
Find out how Sarepta’s 3-year Elevidys trial data could reshape investor sentiment, regulatory outlook, and Duchenne gene therapy strategy.
Atossa Therapeutics (Nasdaq: ATOS) gains FDA orphan drug designation for (Z)-endoxifen in Duchenne muscular dystrophy
Find out how Atossa Therapeutics’ orphan drug nod for (Z)-endoxifen in Duchenne muscular dystrophy could reshape its R&D roadmap.
AGAMREE wins Swissmedic approval, positioning Santhera for broader Duchenne muscular dystrophy market expansion
Santhera wins Swissmedic approval for AGAMREE in Duchenne muscular dystrophy. Find out how this milestone could drive European expansion and investor sentiment.
Avidity Biosciences (Nasdaq: RNA) opens access to del-zota in DMD44 as BLA filing nears
Avidity Biosciences launches U.S. access program for del-zota in DMD exon 44 patients. See how this ties into FDA submission plans and the Novartis acquisition.