Fingolimod treatment leads to beneficial effects in Huntington disease (HD), according to a new study in a mouse model of the ...

Alpha-1 antitrypsin deficiency (AATD) is found to be associated with pregnancy-related complications and adverse birth ...

Overall, 80.9% of patients were in complete remission 1 year after diagnosis irrespective of GPA or MPA cases (79.7% vs 83.8%, respectively). The therapeutic armamentarium for individuals with ...

The navenibart phase 3 program will consist of the ALPHA-ORBIT phase 3 trial and the long-term extension trial, which are designed to support registration globally. Global start-up activities ...

Nusinersen, an SMN2-targeted antisense oligonucleotide drug administered by intrathecal injection, was able to improve motor function and survival. Treatment with nusinersen can improve motor function ...

The adolescent HAE population continue to experience unique challenges to their school and social lives that degrade their health-related quality of life. Adolescents with hereditary angioedema (HAE) ...

Vamorolone at a dose of 2 mg/kg a day was associated with improvements in the velocity of the 4-stair climb test and the supine-to-stand test. A new systematic review and meta-analysis of the ...

In 2025, the company plans to further their SM program by presenting additional results from the PIONEER trial and activating the phase 3 study of elenestinib for indolent SM. Blueprint Medicines ...

These markers could serve as indicators of disease severity and progression, offering potential tools for personalized therapeutic intervention. Neurophysiological and ultrasonographic features can ...

Ocular manifestations of MPA are uncommon, but early recognition may allow for prompt initiation of appropriate treatment to prevent further complications. An elderly man with retinal vasculitis was ...

Vosoritide was approved in 2021, after current achondroplasia guidelines were published, creating a need for treatment and monitoring recommendations. A new international consensus statement has been ...

Analysis of genetic similarities between IPF and GERD, 2 conditions that often coexist, may aid in the selection of therapeutic targets. Nine hub genes have been identified as significantly altered in ...