Regarding the pediatric population, mold-active antifungal prophylaxis is recommended for pediatric patients at high risk of IFD, including a subgroup of children with ALL. Regarding the pediatric ...

In diagnosed cases of cutaneous mastocytosis, investigations to rule out systemic mastocytosis may be appropriate. A young child with a small chest lump and anemia was found to have cutaneous ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

In treatment-refractory EGPA, physicians should initiate additional investigations as soon as possible to identify the therapies best suited to prevent further clinical deterioration. A female child ...

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 ...

Z-rostudirsen is designed to enable production of near full-length dystrophin in both muscle and the central nervous system, unlike similar therapies that target muscle alone. Dyne Therapeutics has ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

The study showed a significant increase in antibody-dependent cellular cytotoxicity for GASDALIE, AFUC, and AFUC GASDALIE variants (40%-49%) compared with wild-type ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Albert Faro, MD, vice president for clinical affairs at the Cystic Fibrosis Foundation (CFF). Dr. Faro speaks about COVID-19’s ...

As winter sets in, many people enjoy a hot mug of cocoa, snuggly sweaters, and outdoor activities such as walking through town to watch the lighting of the town tree or skiing with friends. There are ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...