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HP&M Attorney Mark Tobolowsky Co-Authors Article in Human Gene Therapy Advocating for the Acceptance of Microdystrophin Expression in DMD Patients as a Surrogate Endpoint for Accelerated Approval
March 19, 2023Hyman, Phelps & McNamara, P.C.’s Mark Tobolowsky co-authored the peer-reviewed article “Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials” in the recently published edition of Human Gene Therapy. The article arose out of work conducted by the Pathway Development Consortium, a partnership between stakeholders in the AAV gene therapy space.
The article describes the challenges facing development of AAV gene therapy treatments for Duchenne Muscular Dystrophy, a serious and life-threatening condition. In particular, as the gene encoding the full-length dystrophin protein is too large to fit inside a single AAV vector used to deliver genes to human cells, development has focused on designing shortened versions of the dystrophin gene, known as microdystrophins, that can fit inside the AAV vector and retain the primary functionality of the full-length dystrophin protein. The article describes the evidence supporting microdystrophin expression as a reasonably likely predictor of clinical benefit from AAV DMD gene therapy treatment and advocates for the use of accelerated approval for such therapies, as appropriate.
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- Supreme Court Makes Quick, Unanimous Work of Seventh Circuit’s Interpretation of False Claims Act Scienter Requirement June 8, 2023
- An Offer You Can’t Refuse: Merck Attacks Medicare Negotiation Program as an Unconstitutional Taking June 7, 2023
- AMCP Format for Formulary Submissions Revision – Comment Period Now Open! June 5, 2023
- CCP Improvements Allow Industry to Track Pre-submissions June 5, 2023
- CMS Publishes Grab Bag of Proposed Changes to the Medicaid Drug Rebate Program June 2, 2023
- Big Molecule Watch Blog
- Bloomberg BNA Health Care Blog
- Drug and Device Law Blog
- Eye on FDA
- FDA Matters
- Harvard Law Bill of Health
- IN VIVO Blog
- Internet Drug News.com
- Lachman Consultants Blog
- Medical Devices Today
- Orange Book Blog
- The Orange Book Insights Blog
- Pharma IQ
- Pharmalot
- SCOTUS Blog
- The Volokh Conspiracy
- WLF Legal Pulse
- Advertising and Promotion (Federal Trade Commission)
- Advertising and Promotion (OPDP)
- Animal Drugs and Feeds
- Biosimilars
- Cannabis
- cGMP Compliance
- Consumer Product Safety Commission
- Controlled Substances
- Cosmetics
- COVID19
- Current Affairs
- Dietary Supplements
- Drug Development
- Drug Enforcement Administration
- Enforcement
- FDA News
- Foods
- Foods and Dietary Supplements
- Fraud and Abuse
- Government Pricing
- Hatch-Waxman
- Health Care
- Health Privacy
- Import/Export
- In Vitro Diagnostic Devices
- Jobs
- Medical Devices
- Miscellaneous
- Orphan Drugs
- OTC Drugs and Cosmetics
- Prescription Drugs and Biologics
- Product Jurisdiction and Combination Products
- Reimbursement
- Tissue Products
- Tobacco
- Uncategorized