A note from Debra

The PJ Nicholoff Steroid Protocol

Posted on April 23, 2015 9:53 pm

CureDuchenne Cares is pleased to share important information about steroids use for Duchenne patients. 

 

Use of corticosteroids is part of the standards of care for Duchenne patients. Chronic use of steroids is accompanied by side effects, but rapid withdrawal of steroids can lead to serious, life threatening conditions as well. This was exemplified in the unfortunate story of PJ Nicholoff, a Duchenne patients who had been on steroids for 25 years when he ended up in hospital with several fractures. A combination of factors, including the rapid withdrawal of daily steroids, sadly contributed to his death.  To raise awareness and increase correct action upon hospitalization of patients, a panel of experts in neuromuscular steroid care has developed a protocol to address rapid withdrawal from steroids, and subsequent acute adrenal suppression, which can be a life threatening condition. It is our hope that families and medical providers, armed with this information, can prevent future occurrences of acute adrenal insufficiency.

For more information and to download the “PJ Nicholoff Steroid Protocol,” please click here 

BioMarin Update on Duchenne Muscular Dystrophy Program

Posted on April 13, 20159:35 pm

CureDuchenne is pleased to share the following communication update from BioMarin to the Duchenne community regarding developments of their exon skipping program for exon 51. CureDuchenne has provided extensive support in developing this program throughout the years.  We are delighted that boys will begin redosing.   We would like to update the Duchenne community on Read more »

Self-Stretches for Duchenne

Posted on February 17, 20154:33 pm

  As part of CureDuchenne Cares – a physical therapy, education and outreach program for the Duchenne community – we are pleased to share information on a self-stretching routine for those living with Duchenne muscular dystrophy. CureDuchenne and the Duchenne Therapy Network created a new video blog and a printable handout on stretching. Performing a Read more »