This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296). We applied a mixed-effect model to examine differences in serum NF levels among healthy control infants (n = 13), untreated SMA infants (n = 68), and SMA infants who received the genetic therapies nusinersen and/or onasemnogene abeparvovec (n = 22). Increased NF levels were inversely associated with copy number. SMA infants treated with either nusinersen or onasemnogene abeparvovec achieved important motor milestones not observed in the untreated cohort. NF levels declined more rapidly in the nusinersen cohort as compared with the untreated cohort. Unexpectedly, those receiving onasemnogene abeparvovec monotherapy showed a significant rise in NF levels regardless of copy number. In contrast, symptomatic SMA infants who received nusinersen, followed by onasemnogene abeparvovec within a short interval after, did not show an elevation in NF levels. While NF cannot be used as the single marker to predict outcomes, the elevated NF levels observed with onasemnogene abeparvovec and its absence in infants treated first with nusinersen may indicate a protective effect of co-therapy during a critical period of vulnerability to acute denervation.
DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.message sent
email sent successfully
rareLife solutions 606 Post Road East #397 Westport, CT 06880 |
||
You are receiving this because you have an account on www.oneSMAvoice.com | ||
If you'd like to unsubscribe and stop receiving these emails click here |
rarerelated tag: Standard Therapies
send a message
Reset password
password changed successfully!
please log in with your email address and new password.
your activation key expired
this confirmation key has expired. please try to log in again or resend confirmation email.
confirmation email sent
a confirmation email has been sent to your inbox. click the link in the email to activate your account.
can't find the email? be sure to check your spam folder.
password reset email sent
an email has been sent to you with a link to reset your password.
can't find the email? be sure to check your spam folder.
If you are a doctor or other qualified health care professional, you should not offer any medical advice or treatment on our Sites, nor should you allow the content of our Sites to substitute for your own medical judgment. Please thoroughly review the information provided on our Sites before deciding whether any of the products, services, or treatments therein are right for you or others.
your account is now activated!
Log in
This feature is only available to members.
you haven't confirmed your email address yet. resend confirmation email