Disease Tab: Neuro
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1
FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
The current therapies for drug-resistant epilepsy are only partially effective and have serious drawbacks. One treatment that can significantly reduce seizure frequency is the removal of the affected part of the brain using surgical or laser ablation methods. However, not surprisingly, removal of brain tissue can cause serious, irreversible damage, such as effects on memory, […]
Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke
Gary Steinberg, M.D., Ph.D., and his team at Stanford University are conducting a clinical trial to test a therapy for motor disabilities caused by chronic ischemic stroke. While “clot busting” therapies can treat strokes immediately after they occur (acute strokes), these treatments can only be given within a few hours of the initial injury. There […]
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to loss of limb function, difficulty breathing, paralysis, and eventually death. There are medications that can slow down the progression of ALS, but unfortunately there is […]
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair and The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord. This damage leads to lifelong lower body paralysis, and bladder and […]
A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease
Parkinson’s Disease is a neurodegenerative disease that is thought to be the outcome of the gradual breakdown of dopaminergic neurons in the brain, which are a type of cell that produces a chemical in ones’ brain known as dopamine. This decrease in the brain dopamine content can result in symptoms such as uncontrollable shaking of […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient’s motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells into patients with ALS. When transplanted into the patient spinal cord, these cells become astrocytes, the support cells that keep nerve cells functioning. Due to […]