Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

Duchenne Surprise: Sarepta Prices Exondys 51 Below Expectations

Executive Summary

It sparked at least one analyst to question how Sarepta came up with a price for FDA's first approved Duchenne muscular dystrophy treatment.

You may also be interested in...



Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen

The US FDA issued a complete response letter rejecting golodirsen based on risk of infection at intravenous infusion ports and preclinical renal toxicity.

Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements

The company presented encouraging data on functional assessments of four boys treated with a micro-dystrophin gene therapy at the World Muscle Society, furthering momentum around the program.

Tech Transfer Roundup: Arrakis Gets Penn IP Toward Small Molecule RNA Therapies

Canaan Partners-backed biotech will investigate the therapeutic potential of three-way junctions in folded RNA. UC Berkeley and Duke sign separate license agreements related to CRISPR gene-editing technology.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

SC097320

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel