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 ...

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 ...

US and European regulators could approve up to 17 gene therapies this year, with a top official at the US Food and Drug Administration (FDA) predicting that 2024 will be a “breakout year” in ...

Over 80% achieved a clinically meaningful improvement in at least one of the scales assessed. Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult ...

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 ...

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 ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

Jennifer Miller, MD, a professor of pediatric endocrinology, speaks about her research on Prader-Willi syndrome from her office at the University of Florida in Gainesville. (Photo by Larry Luxner) ...

This comprehensive popPK analysis adds to the pool of knowledge on oligonucleotide PK properties and helps optimize the appropriate use of imetelstat. The nonlinear mixed-effects model identified ...

Ofirnoflast’s NEK7 modulation offers a first-in-class anti-inflammatory approach to MDS. The US Food and Drug Administration (FDA) granted Orphan Drug Designation to ofirnoflast (HT-6184) for the ...