Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

In the FRONTIER3 trial, 74.3% of children receiving once-weekly Mim8 prophylaxis experienced zero treated bleeds. Mim8, an investigational factor VIIIa mimetic bispecific antibody, demonstrated ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease (HD) since 1969. Dr. Lange, a proponent of nondrug ...

Pediatric patients with ALL experience, on average, about 15 different symptoms in their chemotherapy journey. Pediatric patients with acute lymphoblastic leukemia (ALL) experience a wide range of ...

The therapeutic regimen included 2 induction doses of daratumumab within a week, followed by further doses as needed. A combination therapy of daratumumab and romiplostim was successfully used to ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...

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 ...

Given the increased risk of severe organ damage, early consideration of lupus in patients with SCD with atypical presentations is essential. Sickle cell disease (SCD) with concurrent systemic lupus ...

Tenosynovial biopsy obtained during carpal tunnel release surgery in patients with idiopathic carpal tunnel syndrome (CTS) may assist in the timely diagnosis of transthyretin-mediated amyloid ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...

Inherited genetic metabolic disorders, including carnitine palmitoyltransferase II (CPT-II) deficiency, should well be presumed in adults with recurrent events of rhabdomyolysis because of its swift ...

Despite landiolol's efficacy in managing supraventricular tachycardia, it is contraindicated in cases of pulmonary hypertension due to the risk of cardiorespiratory decompensation. The approval is ...