Streamlined Approach to Developing Rare Disease Therapies Being Examined
Initiatives are underway to streamline the pathway to new treatments for rare diseases, a federal health official said at WODC 2023.
SMA News Briefs
Safety and Efficacy Evaluation of Gene Therapy Drug in SMA Type 1 Treatment
The incidence rate of spinal muscular atrophy (SMA) is rising, highlighting the urgent need for more effective treatment options. To combat this, researchers are gearing up for an innovative new clinical trial study that could significantly impact how we approach the treatment of SMA. This new clinical trial sponsored by GeneCradle Inc. is expected to be completed by December…
Muscle Ultrasound Changes Correlated With Functional Status in SMA
A new study published in Ultrasound in Medicine & Biology has revealed that muscle ultrasound changes significantly correlate with functional status and is beneficial for quantifying muscular changes in spinal muscular atrophy (SMA). The study highlighted that the diaphragm thickening ratio could appear normal even with a severe compromise of the respiratory muscles in quantitative analysis. The researchers investigated…
Challenges for Gene Therapy Employing AAVs in Rare Diseases of the CNS
A new review article summarizes the current state of development, as well as the challenges, of gene therapy approaches for rare diseases of the central nervous system (CNS) such as Friedreich ataxia (FA), spinal muscular atrophy (SMA), and Dravet syndrome. The review, published in the Journal of Controlled Release, focuses on the need to overcome…
New Home Monitoring Study on SMA Currently Recruiting
A new clinical trial aiming to compare the results of physical evaluations in hospital and home settings in patients with spinal muscular atrophy (SMA) receiving genetic therapy is currently in the recruiting phase. Therapeutic strategies focusing on the increase of SMA protein production through genetic replacement and premRNA splicing in SMN2 modification with drugs such…
Research Platform May Accelerate Drug Development for PD, SMA, and DMD
A recent study published in the Journal of Neuromuscular Diseases has revealed that neuroMuscular ObserVational Research Data Hub (MOVR) can serve as a potential platform for informing disease understanding, clinical trial design guidance, and accelerating the drug development for neuromuscular diseases (NMDs). According to the researchers, the MOVR platform collects longitudinal data from individuals diagnosed with various…
Clinical Trial on Risdiplam for SMA Will Soon Begin Recruitment
An upcoming study will evaluate the pharmacokinetics and safety profile of risdiplam in patients with spinal muscular atrophy (SMA). Researchers will soon be initiating the recruiting process for this interventional study. This phase 4 clinical trial is part of an open-label study analyzing risdiplam. It will include approximately 10 participants. Read more about SMA therapies…
Clinical Trial Aims to Assess Nusinersen Use in Pregnant Women With SMA
A clinical trial aims to evaluate nusinersen therapy among pregnant women with spinal muscular atrophy (SMA). The upcoming clinical trial sponsored by Biogen Inc began on April 15, 2023. The trial aims to prospectively assess pregnancy complications and outcomes in patients with SMA. The investigators will also look at birth outcomes and adverse effects in infants born to…
New Study Aims to Evaluate Safety and Efficacy of NMD670 in SMA
A new phase 2 clinical trial, called SYNAPSE-SMA, will investigate the safety and efficacy of NMD670 in ambulatory adults with spinal muscular atrophy (SMA) type 3. The study is not yet recruiting but will seek to enroll 54 participants with an estimated start date of May 2023. The study will involve a 2-way crossover design…
Structural Changes Negatively Affect Gross Motor Skills in SMA
A recent study published in Biomedicines has found greater improvement in the upper limb compared to the overall improvement in motor function based on the 12-month observation in patients with spinal muscular atrophy (SMA). The study observed that the more stable older patients showed fewer incremental structural changes than the young ones. Furthermore, steady structural changes negatively…
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