P1-SAF-301 CLINICAL TRIAL

 

 

 

Four patients have been enrolled in the P1-SAF-301 clinical trial and received the investigational treatment in 2011-2012.

Preliminary data, as of date, provide support that the safety profile of the investigational product SAF-301 is acceptable for use in the treatment of patients with Sanfilippo type A syndrome.

PATIENT AREA

 

 

 

As an action of celebration of the World Rare Disease Day 2013, LYSOGENE announces the launch of its new website-section entirely dedicated to Patients, their Families and Patient Organizations (PO’s).

MPS AWARENESS DAY 2013

 

 

 

In order to support the International MPS Awareness Day 2013, LYSOGENE set up different activities targeted to patients and caregivers, medical professionals, researchers, decision-makers and the general public.

We launch a video aimed at communicating about our work and Sanfilippo disease awareness. Additionally, interviews with patient representatives from different countries covering several aspects of their organisations activities were performed.