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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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- FDA Stealthily Convenes Multi-Cancer Testing Panel Meeting November 28, 2023
- Silence Isn’t Golden: Two Executives Convicted in First Criminal Prosecution Under the Consumer Product Safety Act November 28, 2023
- FDA Proposes to Ban Brominated Vegetable Oil in Food November 23, 2023
- Going to 11: New Subsections at CDER’s Office of Pharmaceutical Quality Are Important Piece of FDA’s Inspection and Enforcement Strategy November 20, 2023
- HP&M Recognized by Best Law Firms® as National Tier 1 – FDA Law and Regional Washington, DC Tier 1 – FDA Law (2024 Edition) November 15, 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