Kyle Munz

The extension of the TOPAZ study provided support for the long-term benefits and safety of apitegromab therapy in patients with spinal muscular atrophy (SMA) at 36 months.

A systematic analysis that reviewed screening for sickle cell retinopathy in patients with sickle cell disease (SCD) underscores the importance of advanced imaging measures and further research on screening implementation methods.

Results from a single-center, observational, prospective study suggested that inflammatory markers could predict disease severity and treatment efficacy for patients with spinal muscular atrophy (SMA).

The phase 3, international, open-label, single-group XTEND-Kids study concluded that children with severe hemophilia A younger than 12 years benefited from treatment with efanesoctocog alfa prophylaxis.

The phase 3 PEGASUS trial found that patients with paroxysmal nocturnal hemoglobinuria (PNH) may still experience anemia following recommended therapies, underscoring the need for further research.

Mark Bleackley, PhD, chief scientific officer of Incannex, discusses in further detail the need for a pharmacotherapy like IHL-42X in obstructive sleep apnea (OSA) and the medication's underlying mechanisms.

A cross-sectional study found that static pain measures could be useful for predicting fatigue severity in patients with sickle cell disease (SCD).

A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US.

Part of the Institute for Value-Based Medicine series, the June 20, 2024, event was presented with Banner MD Anderson in Phoenix, Arizona.

Ron Grunstein, MD, PhD, head of sleep and circadian research, Woolcock Institute of Medical Research, details results from the ALKS 2680 trial and the study's next steps.

A retrospective database analysis demonstrated the high clinical burden experienced by patients with severe deficiencies of ADAMTS13 and thrombotic thrombocytopenic purpura (TTP)–related diagnoses.

A multicenter pilot study concluded that dual therapy in spinal muscular atrophy (SMA) does not provide further benefits to infants at risk for SMA type 1.

An investigation into the burden of dilated perivascular spaces (dPVS) in children with sickle cell disease (SCD) confirmed that those with SCD experience more severe manifestations of dPVS compared with the general population.

Mark Bleackley, PhD, chief scientific officer, Incannex, details the progress of the ongoing RePOSA trial, which is assessing the safety and efficacy of IHL-42X in patients with obstructive sleep apnea (OSA).

Michael A. Choti, MD, MBA, Banner MD Anderson, cochaired the second annual Advancing Value-Based Oncology Care meeting and shared insights into the goals of this year's event and value-based medicine as a whole.

Michael Thorpy, MD, professor of neurology, Albert Einstein College of Medicine, discusses the hopeful future of treatment in idiopathic hypersomnia.

Sindhuja Kadambi, MD, MS, University of Rochester Medical Center, discussed in detail the unique considerations necessary for oncology care in the aging population.

Matthew Callister, MD, Banner Health, discusses the advantage of value-based oncology care as a platform for collaboration.

For patients with paroxysmal nocturnal hemoglobinuria (PNH), crovalimab has emerged as an additional treatment option with a similar safety and efficacy profile to eculizumab,

New findings show a similar number of injective-related reactions (IRRs) were reported between the use of ofatumumab (Kesimpta) and ocrelizumab (Ocrevus) in the treatment of multiple sclerosis, but less severe reactions have been associated with ofatumumab.

Results from a cross-sectional study revealed the gaps in knowledge related to fertility risks among patients with sickle cell disease (SCD), as well as shed light on patient perspectives on fertility treatments.

Michael Thorpy, MD, professor of neurology, Albert Einstein College of Medicine explores the current treatment landscape of idiopathic hypersomnia and the associated challenges with approving new medication for this condition.

A 2-sample Mendelian randomization study found a potential connection between dietary intake and multiple sclerosis (MS) risk and severity.

An analysis conducted on UK Biobank data suggests a possible association between higher concentrations of serum albumin and a reduced risk of multiple sclerosis (MS).

A prospective study concluded that patients with narcolepsy can safely switch from a high-sodium oxybate to a low-sodium oxybate to manage their symptoms.

Sickle cell trait (SCT) and pregnancy each exacerbate the symptoms of COVID-19. As a result, pregnant individuals with SCT may have a higher risk of COVID-19 infection.

A poster presented at the 2024 American Society of Clinical Oncology annual meeting argued the benefits that Medicaid expansion can bring to patients with melanoma.

A prospective cohort study, using data from the UK Biobank, suggested that the Mediterranean diet could provide benefits to patients with multiple sclerosis.

June 19 is marked as World Sickle Cell Awareness Day, granting the opportunity to reflect on the current state of treatment, access, equity, and more that impacts patients living with sickle cell disease.