This is the first report from SveATTR— a longitudinal Swedish web-based registry open for TTR variant carriers and patients with ATTR amyloidosis. The study summarizes 60 years of data collected as ...

The chronic use of a TGF-β-targeting phosphorodiamidate morpholino oligomer (PMO) and dystrophin exon-skipping PMO reduced muscle fibrosis and loss, restored dystrophin expression in skeletal and ...

The majority (90%) of neurologists were aware of 40 out of 61 recommendations on the treatment of myasthenia gravis (MG). Most Spanish neurologists know, agree on, and apply guidelines for the ...

Patients with autoimmune nodopathy more frequently had blood–cerebrospinal fluid barrier dysfunction and total intrathecal immunoglobulin G synthesis than patients with chronic inflammatory ...

Myasthenia Gravis Activity of Daily Living (MG-ADL) is able to capture more symptoms than Quantitative Myasthenia Gravis (QMG). Myasthenia Gravis Activity of Daily Living (MG-ADL) can identify ...

In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...

The total symptom score demonstrated excellent internal consistency (Cronbach’s alpha =0.84-0.92) and strong test-retest reliability (intraclass correlation coefficient =0.86-0.97). The Mastocytosis ...

Transthyretin amyloid deposits were observed mainly in the vessel walls of various organs throughout the body in all autopsy cases with Alzheimer's disease and ATTR-CM. Transthyretin (TTR) amyloid ...

There are significant differences in terms of survival between non-anemic patients with MF, those with nontransfusion-dependent anemia, and those with transfusion-dependent anemia. A team of ...

Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...

The primary outcome measures include assessing I-RODS scores at baseline and monitoring variations at 6 and 12 months after enrollment An upcoming observational, noninterventional study aims to assess ...

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...