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

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 ...

There were fewer multiorgan transplants in patients with hereditary ATTR-CM with increased use of disease-modifying therapies, a study found.

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) ...

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

The overall survival rate at 18 months for patients diagnosed before the introduction of the new guideline was 79% while it was 90% after the introduction of the guideline. The number of patients ...

rhG-CSF has a short half-life and requires daily injection to maintain an effective blood concentration. Pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) reduced the ...

Dr. Bichell is part of a panel studying how the US FDA can speed up clinical trials and be more transparent about its decisions. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

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 ...