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 |
Trusted Resources: News & Meetings
Latest announcements and gatherings
Gene Therapy Improves Muscle Function for Children With Spinal Muscular Atrophy
Children with spinal muscular atrophy (SMA) carry specific gene defects which cause progressive muscle wasting and weakness. Left untreated, the condition usually results in early death. Approval was recently granted for a gene replacement therapy that enables the maintenance of muscle function. However, due to the treatment’s relative novelty, data on safety and efficacy remain limited.
A team from Charité – Universitätsmedizin Berlin recently conducted the first multicenter clinical study to evaluate data on the side effects associated with this new gene replacement therapy. Their study showed that treatment resulted in significant improvement in muscle function, particularly in children under two years of age. The researchers’ findings have been published in The Lancet Child & Adolescent Health.


Related Content
-
EducationImplications of Circulating Neurofilaments for Spinal Muscular Atrophy Treatment Early In Life: A Case SeriesThis longitudinal cohort study aimed to ...
-
News & MeetingsSTR1VE-EU Trial Shows Efficacy of Onasemnogene Abeparvovec in Spinal Muscular AtrophyOnasemnogene abeparvovec gene replacemen...
-
News & MeetingsNewborn Screening, Treatment Make Big Difference With Rare Muscle DisorderMateo Medina has never walked or talked....
-
News & MeetingsWill Parental Vaccine Hesitancy Retard the Embrace of Life-Saving Newborn Genetic Screening and Emerging Gene Therap...In these days of the never-ending pandem...
-
People & PlacesAnne M. Connolly, MDAnne M. Connolly, MD, is the Chief of th...
-
People & PlacesMegan A. Waldrop, MDMegan Waldrop, MD, is a Pediatric Neurol...
-
EducationGuidance on Gene Replacement Therapy in Spinal Muscular Atrophy: A Canadian PerspectiveSpinal muscular atrophy (SMA) is the sec...
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