Liftstream kindly provided this whitepaper entitled, The Path to New Therapies: Duchenne Muscular Dystrophy.
In the whitepaper, Liftstream “explores four of those companies driving different therapeutic approaches for DMD: Sarepta and Prosensa, who are both advancing exon skipping therapies, PTC Therapeutics who is developing nonsense mutation (nmDMD) and Summit Corporation PLC who is exploring utrophin modulation.”
The report outlines major hurdles for new clinically proven therapies, including the regulatory approval process, manufacturing challenges, pricing, reimbursement and market access.
If you want to know more about:
- The patient perspective
- Novel therapeutic approaches for DMD
- Exon skipping
- Utrophin modulation
- After clinical trials
- Access to medicine
- Market access stakeholders
- Manufacturing challenges in rare diseases
- Patient registries
- Aligning stakeholders in rare diseases
…then you must download the whitepaper now!
Lifstream is an associate sponsor of the upcoming World Orphan Drug Congress USA, taking place this April in Washington, DC.
Marketing Director for Terrapinn’s Americas business, Caroline Hornby currently brings patient groups and industry together at World Orphan Drug Congress USA.
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