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.
Neurologists
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…
A Rare Case of Pompe Disease and Congenital Myasthenic Syndrome
Researchers reported a rare case of a 4-year-old girl with co-occurrence of Pompe disease and congenital myasthenia syndrome type 5. The case report, published in Cureus, described good results from whole-exome sequencing for an early diagnosis and subsequent alglucosidase alfa enzyme replacement therapy (ERT), which is currently the only approved treatment for Pompe disease. “Early…
Rituximab Effective for MG but May Cause Severe Infection
Rituximab is effective in treating myasthenia gravis (MG), especially in patients with antibodies to muscle-specific kinase (MuSK), however, may increase the risk of severe infections, according to a study published in Neuromuscular Disorders. During the study, all 12 patients with anti-MuSK antibodies achieved minimal manifestation status (MMS) or remission, but only 33% (6/18) of patients…
Longer Walk Distance Correlated With Improved Outcomes in Pompe Study
Scientists have discovered that improved walking distance is associated with improved quality of life in patients with Pompe disease, and published their findings in Value in Health. In incurable, progressive diseases such as Pompe disease, improving a patient’s quality of life becomes the cornerstone medical objective. Scientists have explored various ways to measure the quality…
Cardiac Autonomic Dysfunction Linked With Parasympathetic Impairment in MG
Researchers in Poland explored cardiovascular autonomic dysfunction in patients with myasthenia gravis (MG) by utilizing various testing methods and published their results in the Journal of Clinical Medicine. They included 38 patients with MG whose cardiovascular autonomic testing results were compared with those of 30 age- and gender-matched controls. The study recorded participants’ cardiovascular parameters,…
Researchers Compare Best Surgical Approaches for Treating MG
Right unilateral thoracoscopic thymectomy and thoracoscopic subxiphoid thymectomy have advantages over transsternal thoracoscopic thymectomy to treat nonthymomatous myasthenia gravis (MG) in terms of short-term recovery following the operation, according to a new study published in Updates in Surgery. However, the transsternal approach “is still the best choice for ectopic thymectomy while thoracoscopic subxiphoid approach show…
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