The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families. Photographs are for illustrative purposes only.
Following an initial increase and peak in motor skills from baseline, the subsequent downturn marks the onset of SMA, with disease progression (and loss of motor skills) being most rapid in the early stage compared to the later stage.1 Several motor-function scales have been developed that are useful in quantifying the natural history of SMA, as well as the response to investigational therapeutic agents in clinical trials.2-4
There are very few books in existence that could explain what SMA means for children living with the disease. The story of Zac’s play day has been written to help children with SMA better understand their condition and learn how to explain it to their friends.
The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families.
Photographs are for illustrative purposes only.
At Biogen, we are committed to support HCPs in their clinical practice. Check out the point of view of some of your peers:
Together in SMA was created in support of that unity, and with the goal of becoming a valued tool for members of the team. Here you’ll find clinical information and care strategies that some people use to help manage the disease.
Biogen is building on our legacy of rare disease research and development to explore potential treatment options. And even then, we’ll still be together in SMA.
1. Swoboda KJ, et al. Perspectives on clinical Trials in Spinal Muscular Atrophy. J Child Neurol 2007;22(8):957-966.
2. Glanzman AM, et al. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord 2010;20(3):155-161.
3. De Sanctis R., et al. Developmental milestones in type I spinal muscular atrophy. Neuromuscular Disorders, Volume 26, Issue 11, 2016, 754-759.
4. Mercuri E, et al. Patterns of disease progression in type 2 and 3 SMA: implications for clinical trials. Neuromuscul Disord 2016;26(2):123-131.