The devices are available for patients aged 17 years and older at no extra cost and can be used after receiving training from a healthcare professional. Two new medical devices that will facilitate ...

(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) ...

Frank Rivera, president of Stronger Than Sarcoidosis, speaks at the 2024 World Orphan Drug Congress USA in Boston, Massachusetts (Photo by Manuela Callari, PhD) Prolonged uncertainty about the future ...

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 ...

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 ...

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 ...

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 ...

All patients selected the mid-response option, which was “some problems” for mobility. The mobility item of the Euro-Quality of Life 5 Dimension 3 Level survey (EQ-5D-3L) has limitations in patients ...

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 ...

New research indicates that obinutuzumab is a potentially effective and cost-effective treatment for patients with rituximab-resistant AAV. Obinutuzumab therapy demonstrated immunological superiority ...

Adjusted analysis found no statistically significant difference in grade 3+ hematologic adverse events between the older and younger cohorts. Standard frontline rituximab, cyclophosphamide, ...

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, ...