Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.
Dyne Therapeutics is preparing a U.S. Accelerated Approval submission for z-rostudirsen in Duchenne muscular dystrophy and gearing up for a Phase 3 trial of z-basivarsen in myotonic dystrophy, while scaling operations to support potential commercialization.
Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.
Monthly patent reviews from April through December 2025 highlight notable drug discovery IP disclosures across targeted protein degradation, small molecule modulators, and emerging therapeutic targets in oncology, immunology, and neurology.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07416929 |
Manual Therapy and Inspiratory Muscle Training in Neuromuscular Disease |
COMPLETED | NA |
| NCT07357428 |
Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI) |
RECRUITING | NA |
| NCT07331025 |
Ultrasound Detection of Early Facial Muscle Changes in FSHD: Thickness and Echo Intensity Findings |
COMPLETED | |
| NCT07121075 |
The Effects of a Dynamic Arm Support in Daily Life |
RECRUITING | |
| NCT07101978 |
Hyperthermia in Patients With Chronic Primary Pain - Effects on Thermoregulation, Somatosensory System and Movement Evoked Pain |
NOT_YET_RECRUITING | NA |
| NCT07071935 |
A Clinical Trial of Early Ventilation in Amyotrophic Lateral Sclerosis (EVENT ALS) |
NOT_YET_RECRUITING | PHASE2 |
| NCT06866483 |
Validation and Scaling of Screening Program for Undiagnosed Myasthenia Gravis-Social Media Campaign Paired With a Self-moderated Assessment |
RECRUITING | |
| NCT06859229 |
New Technological Pathway for Gait Rehabilitation |
NOT_YET_RECRUITING | NA |
| NCT06791369 |
The Prevalence of RYR1-related Disease |
NOT_YET_RECRUITING | |
| NCT06692660 |
Overview: Use of Respiratory Support At Home in Children |
NOT_YET_RECRUITING |
