duchenne Ireland - Research

By: Duchenne Ireland  05/12/2011
Keywords: Clinical Trials

September 2011

Genzyme has developed this Programme Update as a way to communicate about

ataluren and as part of our ongoing commitment in keeping the DBMD community



As reported in our June Programme Update, Genzyme has been actively exploring

various options in our efforts to determine appropriate next steps for the nmDBMD

programme. While we have been very pleased with our long-standing relationship

with PTC, as a result of our recent portfolio alignment process, we would like to

inform the community that Genzyme has decided to restructure our relationship with

PTC for the ataluren nmDBMD programme. We will, however, retain an option to

reengage the collaboration for non-nmDBMD indications, which reflects our belief in

the potential of this approach for the treatment of nonsense mutation genetic


Also in our June Programme Update, Genzyme informed the nmDBMD community

that we initiated start up activities for a follow-on clinical study for patients who

participated in earlier ataluren nmDBMD clinical trials.  Moving forward, PTC now has

full, global responsibility for the clinical, regulatory and commercial strategy of

ataluren in nmDBMD. Genzyme will work closely with PTC to ensure an efficient

transition of all DBMD related activities to PTC. Questions relating to the follow-on

safety study follow-on clinical study for patients who participated in earlier ataluren

nmDBMD clinical trials should be directed to PTC. 

If you have any questions relating to this announcement or the follow-on safety study,

we would kindly ask that you forward these directly to PTC who are best placed to

address them. PTC can be contacted either by e-mail at

or by telephone +1 866 282-5873.

Genzyme is deeply impressed by the unwavering dedication, support and

collaboration within the community on the behalf of families affected by DBMD,

particularly the boys living with this disease. We have greatly appreciated the

community’s patience and engagement in ongoing dialogue during our involvement

with the ataluren nmDBMD programme. Even though Genzyme will no longer be

involved with ataluren in nmDBMD, we would like to assure you that we remain

committed to the neuromuscular patient community and will continue to look for

innovative, transformative therapies for those with unmet medical needs.

Keywords: Clinical Trials

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