Cardiologists

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…

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,…

Next post in LCFAOD Insights