Proactive management of skin reactions to mirdametinib, a treatment for NF1-associated plexiform neurofibromas, may improve ...

A Finnish cohort study showed that patients with acromegaly had a 59% higher hazard of cardiovascular morbidity than matched ...

Clinicians in India reported what may be the first case of FCS caused by a homozygous LMF1 variant diagnosed in infancy.

Treatment with antenatal IVIG and steroids protected a second sibling from developing the bleeding symptoms and other ...

The global phase 2 T-RRex study has opened an MRD-positive cohort evaluating Sofi-cel in patients with T-ALL/T-LBL.

The earlier onset of PPF is associated with a higher risk of mortality in patients with CTD-ILD, according to a recent study.

Even when a treatment plan works to prevent NMOSD relapses, the regimen can take a mental and physical toll on the patient.

Patients with IPF treated with inhaled treprostinil at 9 breaths per session or more showed greater improvement in forced ...

Since the adoption of newborn screening for SMA, onasemnogene abeparvovec has become the most common first-line therapy in US infants.

Increased emphasis has focused on parent-led and other nonpharmacologic approaches intended to support the well-being of ...

Aarush approached Veer with a novel concept: using AI to create a public, searchable free platform built exclusively for rare diseases.

Deutetrabenazine significantly reduced chorea severity over 16 weeks in a prospective study of Chinese adults with Huntington disease (HD).