Prophylaxis With Recombinant ADAMTS13 Demonstrates Superiority vs Standard Therapy in Congenital TTP
The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with congenital thrombotic thrombocytopenic purpura (cTTP) compared with standard therapy.
Immunological Events Indicated in Onset of Hypersomnolence Disorders, Study Finds
Findings from a retrospective study suggested a possible causal relationship between prior infection and the development of hypersomnolence disorders such as narcolepsy and idiopathic hypersomnia, but more research is needed.
Pegcetacoplan Contributes to Better Outcomes in PNH vs Other Treatments
An analysis of 2 phase 3 trials supporting the use of pegcetacoplan (PEG) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) assessed the benefits of this treatment compared with eculizumab (ECU) and supportive care measures.
Ryplazim Marks First FDA Approval for Plasminogen Deficiency: Dr Amy Shapiro
Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, continues her discussion on plasminogen deficiency type 1 by highlighting the recent FDA approval of plasminogen (Ryplazim; Kedrion Biopharma), the first treatment specifically indicated for this disorder.
Serum Neurofilament Light Changes Not Always Apparent in Active RRMS, Study Finds
A prospective study found evidence of serum neurofilament light (sNfL) level increases in patients affected by active forms of relapsing-remitting multiple sclerosis (RRMS); however, these findings were not significant enough to suggest sNfL measurements replace clinical or MRI monitoring of disease activity.
Exagamglogene Autotemcel Shows Promise in Treatment of Transfusion-Dependent β-Thalassemia
An ongoing, phase 3, open-label study demonstrated the efficacy of exagamglogene autotemcel (exa-cel) infusion to promote transfusion dependence in patients with transfusion-dependent β-thalassemia (TDT).
Dr Kathy Zackowski Discusses the Importance of Rehabilitation Research and Trials in MS
Kathy Zackowski, PhD, National MS Society, expresses the inherent value of quality rehabilitation trials for broadening clinical understandings of multiple sclerosis (MS) and bettering patient outcomes.
Dr Dalia Rotstein: Physicians Must Be Aware MS Affects People of All Backgrounds
Dalia Rotstein, MD, MPH, emphazises the importance of awareness that multiple sclerosis (MS) impacts patients from various backgrounds as clinicians think through ways to improve access to care and research efforts in MS.
Dr Diana Isaacs Surveys Emerging Diabetes, Obesity, and Cardiometabolic Treatment Landscape
Diana Isaacs, PharmD, chair of a recent Institute for Value-Based Medicine® event hosted in conjunction with Cleveland Clinic, details the up-and-coming therapies impacting the scope of treatment in diabetes, obesity, and cardiometabolic health, as well as their associated challenges.
Dr Robert Zimmerman Discusses Challenges, Adverse Effects of GLP-1 and Multi-Agonist Therapies
Robert Zimmerman, MD, discussed the adverse side effects associated with glucagon-like peptide-1 (GLP1) and multi-agonist therapies, as well as explored the challenges in developing these interventions.
Award-Winning Poster Presentations From AMCP 2024
At the Academy of Managed Care Pharmacy (AMCP) 2024 annual meeting, multiple poster presentations concerned with health equity, data collection, glucagon-like peptide-1 agonists, and more were acknowledged for their originality, relevance, clarity, bias, and quality.