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
UsernamePublicRestriction

Sarepta’s Gene Therapy Impresses In Limb-Girdle Muscular Dystrophy

Pivotal Study Planned For 2021

Executive Summary

Positive functional results from Sarepta’s SRP-9003 gene therapy in limb-girdle muscular dystrophy type 2E bodes well for a pivotal study, and for the company’s studies of other potential gene therapies in Duchenne muscular dystrophy.

You may also be interested in...



Hansa Raises $121m To Fund Idefirix Launch

Having recently signed a gene therapy collaboration with Sarepta and received a positive opinion from the CHMP for its kidney transplant therapy, Sweden's Hansa has now raised a substantial sum in a "multiple times oversubscribed" placing.

Stockwatch: Age And Outcome Duration Cloud Innovative Therapy Reimbursement

Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.

Pfizer Planning To Start Phase III DMD Gene Therapy Trial, With Safety Monitoring Protocols

Updated Phase Ib data showed safety issues due to complement activation, but Pfizer is confident the benefits outweigh the risks and monitoring systems will help as it moves into Phase III.

Topics

Related Companies

UsernamePublicRestriction

Register

LL1134043

Ask The Analyst

Please Note: Click here for more information on the Ask the Analyst service.

Your question has been successfully sent to the email address below and we will get back as soon as possible. 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