The coadministration of D156844 and AR42 leads to an additive improvement in survival in a model of spinal muscular atrophy (SMA), a study found.

Surgery for patients with achondroplasia should involve careful preoperative preparation and the use of specialized positioning equipment.

Patients with Wilson disease are at risk of osteoporosis and osteopenia, particularly older, male patients and those with skeletal symptoms.

Serum transthyretin levels appear to correlate with worse prognosis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a study found.

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 ...

A new study will evaluate the long-term safety and tolerability of HRS-5965 in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Claire Booth, MBBS, PhD, cofounder of the AGORA Initiative.

Patients with gMG still have many unmet needs including lack of access to specialist care and delays in diagnosis and treatment, a study found.

A rare case of coexisting diffuse large B-cell lymphoma (DLBCL) and classical Hodgkin lymphoma presents in a 60-year-old female patient.

Jaycee Knox lives with severe hemophilia, which means his body makes less than 1% of factor VIII, a necessary clotting component to stop and regulate bleeding. He undergoes regular infusions at home ...

In June 2024, the US Food and Drug Administration (FDA) approved Vyvgart ® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc; Argenx BV) for the treatment of adults with chronic inflammatory ...

In observance of Rare Disease Day, Rare Disease Advisor has curated this collection of content to illustrate the issues facing the rare disease community, highlight the advocacy work being done in the ...