Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

A diagnosis of ATTR-CM in a more recent era was associated with older age, White race, wild‐type disease, and tafamidis treatment. The survival of patients diagnosed with transthyretin-mediated ...

Generalized pustular psoriasis (GPP) is a rare, severe form of psoriasis characterized by the acute onset of eruptions of sterile pus-filled skin lesions and rashes with or without systemic ...

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

In a nonhuman primate model of acquired hemophilia A, treatment with PS-siRNA 40031 restored thrombin generation to normal levels. Reducing protein S (PS) levels can increase thrombin generation and ...

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 ...

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 ...

CAD accounts for approximately 15% of AIHA cases 2; various sources indicate that anywhere from 7% to 25% of cases of AIHA are caused by CAD. 3 Secondary CAD occurs as a result of underlying ...

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 ...

Coramitug is well tolerated and leads to a statistically significant reduction in N-terminal pro-brain type natriuretic peptide (NT-proBNP)—a marker of disease progression in transthyretin-mediated ...

A pattern recognition approach takes into account factors like disease progression rate, whether the disease symptoms are symmetrical or asymmetrical, whether they are predominantly distal or proximal ...

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 ...