Foramen magnum development may be largely determined in utero, potentially explaining why postnatal vosoritide was unable to prevent stenosis. Two infants with achondroplasia who initiated vosoritide ...

The model had an area under the receiver operating characteristic curve of 0.83 in a high-risk subgroup of patients with conditions that mimic ATTR-CM. An artificial intelligence model trained to ...

Vesna Aleksovska knows of only 15 other people with Gaucher disease in her country of 1.8 million people. Rare Disease Advisor, a trusted source of medical news and feature content for healthcare ...

Selinexor in combination with ruxolitinib achieved a statistically significant improvement in spleen volume reduction for patients with MF, according to a press release. Rare Disease Advisor, a ...

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 ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

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 ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

In the FRONTIER3 trial, 74.3% of children receiving once-weekly Mim8 prophylaxis experienced zero treated bleeds. Mim8, an investigational factor VIIIa mimetic bispecific antibody, demonstrated ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...

(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...