Given the results, the investigation of intranasal carbetocin in PWS has been halted. Intranasal carbetocin did not demonstrate clinical benefit for hyperphagia in patients with Prader-Willi syndrome ...
In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...
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 ...
Kine Sciences advanced KINE-101 to the phase 1b/2a study following the successful completion of a phase 1 study in the United States. The first patient in a new phase 1b/2a clinical trial to ...
Immunohistochemistry can identify concomitant SM in acute myeloid leukemia, leading to better risk stratification and management. The most common forms of AML in pediatric patients are those ...
A patient presented with task-related isolated transient weakness in her right hand, which started 15 years before and lasted for 9 years without worsening or improvement. Distal myasthenia gravis (MG ...
In addition to improved functional AAT levels in the lungs, inhaled AAT demonstrated good safety and tolerability. Patients with alpha-1 antitrypsin deficiency (AATD) receiving inhaled ATT therapy ...
Inherited genetic metabolic disorders, including carnitine palmitoyltransferase II (CPT-II) deficiency, should well be presumed in adults with recurrent events of rhabdomyolysis because of its swift ...
CAD secondary to SLE most often presents at the onset of lupus, but it occasionally develops within the first 5 years. Although rare, secondary cold agglutinin disease (CAD), a type of autoimmune ...
The levels of NT-proBNP, left ventricular mass index, maximal wall thickness, and E/E′ ratio were higher in patients with worse neurological stages compared to those in the early neurological stages ...
In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...
Arrowhead Pharmaceuticals announced the first patient treated in Takeda’s phase 3 REDWOOD trial of fazirsiran (TAK-999/ARO-AAT) for patients with alpha-1 antitrypsin deficiency (AATD)-associated liver ...
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