Roche is developing the new generation of treatment for spinal muscular atrophy. With its molecule RG7916, which is administered as a syrup, it is painless as a lumbar puncture. This molecule overcomes the blood-brain barrier. This drug has the advantage,it reaches both the organs and tissues in the body as well as the central nervous system. RG7916 was well tolerated by the patients and no serious side effects.
It’s a heart-breaking situation and one Kristina Gelblin, Patient Support Partner at Roche, is all too familiar with. Kristina is the vital link between patients, their families and the research centres at the forefront of the race to find new treatments for spinal muscular atrophy. Here, we tell the story of one baby recently enrolled in one of our research studies.
“Before a family can relocate to another country with their child to participate in the clinical trial a lot of different things have to be arranged,” says Kristina. “This includes things like visas and passports, but also their study centre needs to speak to their local treating physician to make sure that this child is really fit for travel and can participate in the clinical trial.
“This is a very challenging situation for the families,’’ she adds. “They have not only just received a diagnosis for the child, but are leaving also their home country, their friends, their jobs and their home to move to another country to participate as quickly and early as possible in a clinical trial.”