Browsing Tag
Duchenne muscular dystrophy
28 posts
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.
Atossa Therapeutics (NASDAQ: ATOS) expands (Z)-endoxifen to DMD: Will investors buy the thesis?
Atossa Therapeutics eyes new ground in DMD care with (Z)-endoxifen. Find out how this small molecule could reshape treatment for carriers and boys alike.
Why the FDA’s warning on Elevidys matters for Duchenne muscular dystrophy treatment
Sarepta’s Elevidys gene therapy now carries an FDA boxed warning. Find out what it means for Duchenne patients, investors, and gene therapy safety.
Sarepta Therapeutics (NASDAQ: SRPT) hit by FDA boxed warning as ELEVIDYS loses non‑ambulatory DMD indication
Sarepta’s ELEVIDYS gene therapy label now includes a boxed liver warning and removes non-ambulatory DMD use. Explore the safety updates and investor outlook.