My Diagnosis Story and Life Ahead With hATTR
My journey to a diagnosis of hATTR amyloidosis was long, confusing, and wrought with uncertainty.
Diagnosis & Disease Information
A Study Will Address Muscle Strengthening Techniques for DMD
An upcoming study will evaluate the effects of trunk-oriented exercises compared to whole-body vibration as therapeutic options for patients with Duchenne muscular dystrophy (DMD). Researchers will soon initiate the recruiting process for this interventional study. The clinical trial will include approximately 30 participants previously diagnosed with DMD with randomized allocation and parallel assignment. Read more…
Study Reveals Factors Associated With NMOSD Misdiagnosis
Factors such as a delay in performing a magnetic resonance imaging (MRI) study, the presence of nonneurological symptoms, and a negative aquaporin 4 (AQP4) IgG test could contribute to a late neuromyelitis optica spectrum disorder (NMOSD) diagnosis detrimental to the health of the patient, according to a recently published study in Multiple Sclerosis and Related Disorders.…
The Brains of Patients With MS and NMOSD Are Damaged in Different Ways
There are distinct patterns of structural damage and reorganization in the brains of patients with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD), found a new study published in the journal Multiple Sclerosis and Related Disorders. According to the authors of the study, these findings could help differentiate between the 2 diseases. To explore…
EXPLORE44 Clinical Trial to Investigate AOC 1044 for DMD Now Recruiting
A new phase 1/2 clinical trial, EXPLORE44™ (NCT05670730), to investigate the use of AOC 1044 in healthy adult volunteers and patients with Duchenne muscular dystrophy (DMD) is now recruiting participants at a study location in San Antonio, Texas. The trial, sponsored by Avidity Biosciences Inc, will enroll patients during 2 different parts of the trial.…
Lung Function Correlates With Physical Activity in DMD
There is a good correlation between lung function and the function of the skeletal muscle in the arms and legs in patients with Duchenne muscular dystrophy (DMD), found a new study that appeared in the journal Pediatric Pulmonology. This is the first study that explores the link between functional measures assessing skeletal muscle disease and…
Our Patient Columnist With MG Reflects on Making Goals
Myasthenia gravis (MG) can vary from day to day and year to year.
Strategies for Diagnosing and Treating Hereditary Transthyretin Amyloidosis
The accurate diagnosis and multidisciplinary treatment of hereditary transthyretin amyloidosis are central to improving patients’ quality of life.
Test Your Knowledge About the Risk of Complications in MG
Which of the statements below about the risk of complications in myasthenia gravis (MG) are correct? Select one of the options for each question, then submit your answers to see your score and the average score for everyone who has taken the quiz. Read more about MG complications.
Hereditary ATTR-CM Has a Poorer Prognosis Than the Wild-Type Form
Around one-fifth of elderly patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) have a pathogenic TTR variant, according to a study published in the European Journal of Heart Failure. ATTR-CM has gained increasing recognition in recent years, aided by advances in imaging studies, such as cardiac magnetic resonance and scintigraphy with bone tracers. This has coincided with…
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