Clinical Trial for Nonsense Children/Adults

To:  DMD community

PTC Therapeutics Petition for PTC 124
Clinical Trial for Nonsense Children/Adults

Help us save the lives of ALL CHILDREN with Duchenne Muscular Dystrophy.

PTC Therapeutics needs to help ALL children with nonsense mutation

Sign this petition to show your support and to urge PTC Therapeutics to provide a new Open Study for ALL children with nonsense mutation using the drug PTC 124

Duchenne muscular dystrophy (DMD) is a muscle wasting disease affecting children. DMD is the most common lethal muscular dystrophy and the most lethal of the muscular genetic diseases.

At this time there are no treatments or cures. IAbout a third of the cases the disease are diagnosed on the basis of gait abnormalities at the age of 4 and 5 years. By the time a child is 8–10 years of age, deterioration of the child’s muscles make it necessary to use a wheelchair. By the childs early teens, children are wheelchair bound and, in some cases, neurological and cardiac symptoms are evident. The progression of muscle deterioration and advanced clinical symptoms cause children in their late teens, early twenties to suffer respiratory or cardiac failure. Thus, requiring care twenty four hours a day, seven days a week. The ultimate result is death

Less than 10% of the patients present a nonsense mutation and this defect can be treated by an experimental drug, PTC124. This drug belongs to a new class of small molecules discovered by PTC Therapeutics. PTC124 allows ribosomes to bypass the nonsense mutations (the premature stop signals) in mRNA and continue the translation process to make a full-length and functional protein.

PTC Therapeutics is currently in clinical trial phase 2b, worldwide with the drug PTC124. This study will be completed in 2010. The participants must be five years of age and ambulatory. There are a few exceptions where a very few group of children participated in the earlier clinical trials who were not ambulatory or later lost ambulation between clinical trials. As a result of children losing ambulation between the ages of 10- 12 years we now have many children/youngsters and adults excluded from this trial.

The drug PTC124 is believed to be able to treat or possibly cure this type of mutation in Duchenne Muscular Dystrophy patients with a nonsense mutation.

As a result this petition is being circulated to implore/plead with PTC Therapeutics to start a new open study, with children not included in the phase 2b trial. We are asking PTC Therapeutics to test the long term effects of the drug on others who are older and non ambulatory. This study can give information about safety, effects on skeletal, cardiac and respiratory muscles. It is urgent/critical that PTC Therapeutics considers a new open study for the group not included in the ongoing clinical trials. This study we are requesting will provide important data to help all who have nonsense mutation. We can help save those who are more advanced in this disease. This trial can only help to gain more information and knowledge of this drug.

Living with a lethal progressive muscle wasting disease is devastating for the one affected and their families who watch the progression. It is a financial and emotional burden for the families. It is also a financial burden for the community in which the family lives. This drug offers promises for a better future, and quality of life. The waiting period PTC Therapeutics has placed on their clinical trial is 2-3 years. This time frame will cost those living with this disease the quality of their life and in some cases their life.


In August/2008, a federal judge in Newark ordered PTC Therapeutics to provide Jacob Gunvalson, 16, the PTC 124 drug despite the company’s objections stating Jacob does not meet the clinical trial’s criteria. We also implore/plead that PTC Therapeutics does not appeal the judicial decision and Jacob Gunvalson be given the chance to use PTC-124.

We ask PTC Therapeutics to hold up to their original promise to help all children affected by DMD.

All who sign this petition support the ongoing work of PTC Therapeutics. This petition is made on behalf of all the other children who are not included in the ongoing clinical trials.

PTC Therapeutics can be a part of changing research to help all living with DMD and a chance to save their lives.

Sincerely,

The Undersigned

The Clinical Trial for Nonsense Children/Adults Petition to DMD community was created by and written by David Feder (feder2005@gmail.com).  This petition is hosted here at www.PetitionOnline.com as a public service. There is no endorsement of this petition, express or implied, by Artifice, Inc. or our sponsors. For technical support please use our simple Petition Help form.