Biopharmaceutical company AMO Pharma Limited revealed on Thursday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation for its investigational therapy in development AMO-02 for the treatment of congenital myotonic dystrophy
In addition, some of our study participants also expressed a sense of failure or self-blame for their infertility or for giving birth to a child affected by congenital myotonic dystrophy
Neonates of affected mothers who are born with congenital myotonic dystrophy
have generalised hypotonia, often resulting in respiratory failure and sometimes death (8).
Babies born with congenital myotonic dystrophy
have difficulty breathing, and Sophie was on a ventilator from birth.
Her death certificate said she died of cardiac arrest, pneumonia, marasmus (progressive emaciation caused by lack of food) and congenital myotonic dystrophy
M2 EQUITYBITES-July 27, 2017-AMO Pharma awarded US FDA's orphan drug designation for AMO-02 for congenital myotonic dystrophy
M2 PHARMA-July 27, 2017-AMO Pharma awarded US FDA's orphan drug designation for AMO-02 for congenital myotonic dystrophy
Audrey, 10, has congenital myotonic dystrophy
fiber-type disproportion - a rare genetic disorder characterized by muscle weakness, slow reflexes and low endurance.
Many residents of the northern Los Angeles County community rallied to the couple's defense after authorities accused them of abusing their 15-year-old daughter, Lindsay, who suffered from a condition called congenital myotonic dystrophy
and died at age 15, weighing less than 50 pounds.
Congenital myotonic dystrophy
is a rare form of the disorder occurring almost exclusively in infants of mothers with the adult form of the disease.
AMO's newly awarded investigational therapy AMO-02 is in development for the treatment of congenital myotonic dystrophy
Noting that her death certificate listed cardiorespiratory arrest, pneumonia, marasmus (progressive emaciation caused by lack of food), and congenital myotonic dystrophy
, he said, ``Each one of them by itself is able to cause her death.