AMO Pharma has announced results from its ongoing phase II study investigating two doses of tideglusib (AMO-02) in adults with congenital and juvenile onset myotonic dystrophy. The subjects had an average age of 22 years – six of whom had congenital-onset myotonic dystrophy – and they underwent once daily oral treatment with 1000mg AMO-02. The results showed that treatment was generally safe and well tolerated, and no subjects discontinued treatment due to adverse events or o

On Friday we received the news that Spinraza - the latest (and first) treatment for spinal muscular atrophy - had been approved by the EMA for use in all types of SMA across Europe. Here's our catch up chat on the subject, and you can read the full EMA report here: https://www.muscleowl.com/single-post/2017/04/21/SPINRAZA-APPROVED-EMA #muscleowltalks #peterduffy #michaelahollywood #spinraza #science #research #sma

We continue to see a large increase in the number of drugs progressing through clinical trials and coming to market. Will this translate into treatments for adults as well as children? Let's discuss... #markchapman #michaelahollywood #drugs #treatment #duchenne #science

Following widespread criticism for its price hike in its recently purchased Duchenne drug, Deflazacort, Marathon have agreed terms to sell the $89,000-a-year treatment to PTC Therapeutics, for $140 million in cash and stock. Marathon's press release reads as follows: All Members of the Duchenne Community, today marks an important milestone in the effort to bring EMFLAZATM (deflazacort) to patients age five years and older in the Duchenne Muscular Dystrophy community. We are p

The European Medicines Agency has granted Orphan Drug Designation to Resolaris (ATYR1940) for the treatment of all types of limb girdle muscular dystrophy (LGMD) & Facioscapulohumeral MD (FSHMD). In muscular dystrophies like LGMD, inflammation contributes to muscle damage. The aim of the protein Resolaris is to reduce inflammation by altering the body’s immune response. aTyr Pharma recently reported its phase Ib/II trial for Resolaris, testing it in patients with LGMD 2B, whi