duchenne muscular dystrophy
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FDA gives thumbs down to Sarepta’s next Duchenne’s muscular dystrophy drug
The decision stood in contrast with the agency’s highly controversial approval of Sarepta’s previous DMD drug, Exondys 51. The company’s shares fell sharply in after-hours trading.
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Pfizer gene therapy shows early efficacy in Duchenne muscular dystrophy, but data raise questions
Data from the drugmaker’s Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Audentes, Nationwide Children’s to develop gene therapy in Duchenne muscular dystrophy
An analyst wrote that the therapy’s combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.
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Can CRISPR deliver real progress in Duchenne muscular dystrophy?
After two painfully incremental drug approvals, the Duchenne muscular dystrophy field is still searching for a therapy that can substantially improve patients’ lives and lifespan. Sounds like a job for CRISPR/Cas9.
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5 reasons for the latest drug cost firestorm, this time over Emflaza’s price tag
Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices
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Four days later, Marathon postpones launch of $89K corticosteroid
Marathon Pharmaceuticals is doing some backtracking after the public, the media and now Bernie Sanders objected to the list price of its upcycled corticosteroid for Duchenne muscular dystrophy.
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FDA approves Duchenne muscular dystrophy drug
The FDA has given the green light to Emflaza (deflazacort), a new drug for Duchenne muscular dystrophy. Announced Thursday, the news is bittersweet. Deflazacort is a corticosteroid, a class of drugs that doctors have used off-label for many years.
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FDA greenlights Sarepta Therapeutics’ milestone treatment for Duchenne muscular dystrophy
Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.
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Morning Read: BioMarin pulls plug on $680M Duchenne drug, Valeant’s Pearson cashes in
Plus, health insurance startup Oscar to build concierge clinic in Arizona, AIDS Healthcare Foundation wants the FDA and Congress to investigate Gilead and eHealth hires its new CEO from Playboy.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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Duchenne Muscular Dystrophy market to shoot up 100-fold in five years
As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, […]
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Catabasis raising $20.4M; modulates multiple disease pathways for Duchenne muscular dystrophy, ALS
Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform […]
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Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
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Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
