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.
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