RESOLARIS - THE CURE FOR FSHD?

As I have mentioned in my previous posts, there are no current approved medical treatments that cure any type of muscular dystrophy.  There is truly exciting news  about a clinical trial for Resolaris, a potential solution for people with facioscapulohumeral muscular dystrophy (FSHD).   FSHD typically starts in the face and upper body and progresses to the lower body.  People with FSHD typically experience debilitating fatigue and chronic pain.   FSHD can be fatal, especially with those who have an early onset.  FSHD is typically diagnosed by the pattern of muscle weakness, DNA testing and other symptoms.

Resolaris is a new drug developed by aTyr Pharama,  a San Diego based company.  Resolaris is derived from protein released into the blood by human skeletal muscle cells.  Resolaris is administered intravenously.  aTyr believes that Resolaris will provide help to people with rare myopathies with excessive immune cell involvement.

The clinical trial is the first administration of a naturally occurring protein derived from physiocrines.  Pysiocrines are a new class of physiological modulators that promote the process of restoring stressed or diseased tissue to a healthier state.  The clinical trial is scheduled for 44 FSHD patients in the European Union.  The trial is designed to evaluate the safety, tolerability, pharmacokinetics and biological activity of adult patients.

For more information about Resolaris please visit the aTyr websitehttp://www.atyrpharma.com.

FSH, FSHMD, FSHD, FACIOHUMEROSCPULAR

This post is about FSH called  faciohumeroscapular) (FSHMD, FSHD or FSH), and it also known as Landouzy-Dejerine, This is a common form of muscular dystrophy impacting approximately 7 out of every 100,000 people.  This form of muscular dystrophy in inherited from one of the parents and effects the skeletal muscles of the face (facio), upper arms (humeral) and the scapula (scapula)

The disease was discovered in 1884 by French physicians Louis Landouzy and Joseph Dejerine. In their research paper of 1886, Landouzy and Dejerine drew attention to the fact that this form of muscular dystrophy ran in family groups.   Although, there are cases where there is no family history of muscular dystrophy.

Symptoms usually develop in early childhood.  Approximately, 95% of the individuals have symptoms are that are apparent starting in their teen years.  This disease advances with progressive weakness to the face, shoulders and upper arms.  It also may impact other skeletal muscles.

FSH is characterized at first by weakness in the facial and shoulder muscles.  Then over a period it can spread to other muscle groups.   Muscle weakness in the pelvis and hips can make it difficult to climb stairs or walk for extended distances.

The person in the picture above is a classic example of FSH.  Note that the weak shoulder muscles tend to make the shoulder blades protrude from the back, a common sign known as scapular winging.  Due to the weakness in muscles of the shoulders and upper arms it can be difficult to raise the arms over the head or throw a ball.