Nonprofit Group Team Joseph Helps Meet the Needs of Patients With DMD
The nonprofit Team Joseph raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
Junction: Neurology
An Interview With Marissa Penrod, Founder of DMD Nonprofit Group Team Joseph
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Marissa Penrod, founder of the Indiana-based nonprofit group Team Joseph.
Nabiximols Oromucosal Spray Fails to Meet Primary Endpoint in MS Trial
Nabiximols oromucosal spray, an experimental treatment for multiple sclerosis (MS) spasticity, failed to meet its primary endpoint in the RELEASE MSS1 (NCT04657666) phase 3 clinical trial, according to a press release from its developer, Jazz Pharmaceuticals. During the trial, patients did not meet the primary endpoint of a significant change in Lower Limb Muscle Tone-6,…
Patients With DMD Show Improved Upper Limb Function on CAP-1002
Capricor Therapeutics reported improvements in skeletal muscle function in nonambulatory patients with Duchenne muscular dystrophy (DMD) who were treated with the experimental drug CAP-1002, according to a press release by the company. These results were recorded from the HOPE-2 open-label extension study. According to the findings of this study, patients reported statistically significant improvements in…
FDA Places Hold on Phase 2 DMD Trial for SRP-5051
Sarepta’s Duchenne muscular dystrophy (DMD) drug candidate SRP-5051 (vesleteplirsen) was placed on clinical hold by the US Food and Drug Administration (FDA) due to a serious adverse event of hypomagnesemia that developed during the MOMENTUM clinical trial. The FDA is now requesting information from the company about all cases of hypomagnesemia. “Patient safety is always…
Two Infants With SMA Successfully Treated With Nusinersen Shortly After Birth
Two infants with spinal muscular atrophy achieved normal motor function after being treated with nusinersen shortly after birth.
SMA Stole My Lifelong Dream: Having and Raising a Child
As a woman with SMA, the part I struggle with is accepting I have been cheated of the opportunity to carry a life inside me.
Valproic Acid Affects Efficacy of Nusinersen in SMA Study
The histone deacetylase inhibitor valproic acid cooperates with nusinersen-like antisense oligonucleotides to promote the inclusion of exon 7 and transcriptional elongation from the SMN2 gene, which means that more functional SMN protein can be made from the SMN2 gene. This approach could increase the efficacy of nusinersen and other antisense oligonucleotides for the treatment of…
Combinatorial Therapy Fails to Counteract SMA Pathology
Scientists have reported that a combinatorial therapy of antisense oligonucleotide (ASO) mediated therapy and low dose SMN with the protective modifier Chp1 is unable to significantly decrease the key pathological features of spinal muscular atrophy (SMA), as published in the Neurobiology of Disease. SMA is a progressive neurodegenerative disorder that often results in early death.…
Fall History Predicts MS Fall Risk in Wheelchair and Scooter Users
Researchers discovered that the previous 6-month fall history predicted fall risk in patients with multiple sclerosis (MS) who used wheelchairs and scooters for mobility, as published in Multiple Sclerosis and Related Disorders. Those with a history of falling had double the risk for future falls, they said. Previous studies on fall risk in the MS…
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