Orpheris works closely with patient advocacy groups in the indications we are focusing on.
For ccALD, the first indication we are targeting with OP-101, we work closely with and seek input from ALDConnect (www.aldconnect.org). We are also working with clinical experts in the treatment of neonatal brain injury as well as other neuroinflammatory diseases.
Resources for Patients and Families:
A Mother's Letter to the FDA: Jean Kelley, Mother of Brian Kelley, a ccALD patient
"Our family has lived with this insidious disease for 20 years. In 1995, at the age of 6, our happy, energetic, athletic, thoughtful, loving, son was diagnosed as follow-up of a head injury. Brian hit a woodpile while sled riding. It was an accident that turned out to be a blessing. Despite a bone marrow transplant Brian progressed rapidly. You cannot fathom, until you live it, how upsetting and painful it is to watch your child lose his abilities and skills, one after another after another, relentlessly. On July 24, 1995, Brian's last words to me were a labored and whispered, "I love you". He last saw me smile at him the following February, when he lost his vision. Next came the spasticity that engulfed his body rendering him dependent on others forever. It was a cavalcade of maladies that in retrospect, gave little time for reflection, only reaction. Problems and losses continue to arise. The many children who struggle daily with no treatment deserve a chance at any improvement in their quality of life."