New Ratings scale positioned to deliver meaningful benefit for clinical research presented at 2019 MDA Clinical and Scientific Conference in Orlando, FL

LONDON and DURHAM, N.C., April 16, 2019 /PRNewswire/ — AMO Pharma Limited (“AMO Pharma”), a privately held biopharmaceutical company focusing on rare, childhood onset neurogenetic disorders with limited or no treatment options, today announced the presentation of a new clinician-completed rating scale to assess features of congenital myotonic dystrophy type 1 (CDM1). An overview of the new rating scale was presented on Monday, April 15^th in a poster presentation titled, “The Development of the Clinician-Completed Congenital Myotonic Dystrophy Type 1 Rating Scale (CDM1-RS)” led by Joseph Horrigan MD, AMO Pharma’s chief medical officer, during The Muscular Dystrophy Association (MDA) Clinical & Scientific Conference at the Hyatt Regency Orlando.

“Standard assessment tools should be used to follow FDA guidance and capture all of the characteristic symptoms of a condition to be useful in clinical trials. To that end, the CDM1-RS rating scale has been evolved from the well validated MDHI and CCMDHI measures developed by professor Chad Heatwole and professor Nicholas Johnson in the hope it can become a widely accepted clinician instrument for CDM1,” said Michael Snape, chief executive officer of AMO Pharma. “The availability of scale to assess CDM1 can expand our understanding of this disorder, streamline the diagnostic process and could provide essential insights to guide future clinical research.”

CDM1 is a genetic disease caused by a large tri-nucleotide expansion repeat adjacent to the DMPK gene. Symptoms usually present shortly after birth and include difficulty with thinking and problem solving, speech impairment, symptoms of autism and weakened muscles. There are currently no approved therapies for CDM1. Patients are often treated with a combination of medical interventions designed to address individual symptom as they arise.

The 12th bi-annual IDMC (International Myotonic Dystrophy Consortium) meeting will be held in Gothenburg, Sweden From June 10-14th of 2019. This is a biannual meeting of the key researchers in the Myotonic Dystrophy Space.. This conference is very scientific generally requiring a masters level or greater science skills in the biological area. There is usually attached a lay conference for DM1 and DM2 for those that want to have some interaction with the Myotonic Dystrophy scientists and glean the latest and greatest from the community. See www.idmc12.org for more information.

Some family members attend just to get a sense of the scientific community and the direction of the research and state of the art in potential therapies.

Registration Fee for non-scientists is about $400 for the full program or $40 for the patient program which is one day. The fees are paid in Swedish currency. The description of the patient day (which will be in Swedish I think) is as follows:

THE PATIENT PROGRAM IS BELOW:

Welcome to a lecture day for you with Dystrophy Myotonika and your relatives

In connection with the international conference on dystrophia myotonika IDMC -12 in Gothenburg, a lecture day will be arranged for the patient group and their relatives. International lecturers from IDMC-12 will be invited together with Swedish lecturers, where the current research situation will be presented. Programs for the day will be available on the conference's website https://idmc12.org/

The lecture day will be held at Dalheimer's house on June 14, 2019. The program starts at 10:00 but with registration and coffee from 9.00. The day ends at 16.00. The cost of the day is 400 Incl VAT (320 excl. VAT) and includes lecture, coffee and lunch.

All prices are listed in sec and excluding 25% VAT.

Myotonic Dystrophy a slowly progressing disease, until it is not slowly progressing. many patients reach a point where they can not go up and down steps anymore. This is a big barrier for many people. There are devices though that will help you up and down stairs. One of these is the Liftcar.

The Liftcar takes some getting used to and requires some skill for the operator to use. You need to tilt and remember to hold the button down. It works well and up and down stairs you can go. Myotonic Dystrophy Patients legs get weak with time. Mobility becomes and issue. But this device can help a lot! The downside is that it is really expensive and insurance does not pay for these type of devices for Myotonic Dystrophy Patients in the USA

Here is a video you can review
https://www.youtube.com/watch?v=QhBhuuoeyd4

Hi All…. You’ve noticed we have been missing for many months!. Our hosting company had a file size issue and when repairing this we had a meltdown of hacking and bad scripts on the site. Now we have repaired it all and are back on line so look for our posts again. we will concentrate on Myotonic Dystrophy and related posts. We try to keep up to date information relating to Myotonic Dystrophy on the site….. and info on DM1 that is not readily available elsewhere.

Richard