Andrew Cox, Pharm.D., MBA

This recent study underscores the fact that biomarkers serve not merely as statistical instruments but as insights into disease biology.

Early combination therapy could translate into fewer downstream hospitalizations and lower costs associated with dialysis and cardiovascular complications.

Patients diagnosed with acquired (autoimmune) hemophilia, a serious and rare bleeding disorder, have high rates of hospital readmissions because of infections and bleeding, as well as high death rates especially among older patients.

Results of a real-world study that included people with hemophilia from 33 countries show that people with hemophilia B, especially those with inhibitors, have the greatest unmet need.

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for Pharmacoeconomics and Outcomes Research (ISPOR) webinar.

The company says it plans to file for accelerated approval later this year.

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.

A systematic review and meta-analysis found some supporting evidence for transdermal anti-inflammatory patches, but the results might be described as, well, patchy.

For people with pain hyperacusis, everyday sounds trigger pain. The condition can lead to social isolation and worse. Treatment options are limited.

Positive results reported for pain from knee osteoarthritis among those who have obesity.

Poor balance and coordination are prominent features of Friedreich’s ataxia (FRDA). Czech researchers found that neuropsychiatric symptoms are also common among patients with FRDA.

Prescription of Neurontin (gabapentin) and other gabapentinoids has increased partly because they are viewed as a safer alternative to opioids.

Laser therapies may help with pain after a root canal, but a review of published studies shows their effectiveness can vary significantly. Further research is needed to identify the optimal approach.

As novel gene therapies emerge, there is a complex interplay among their potential benefits, the burdens they may impose and the risks involved, particularly when treating vulnerable populations such as children.

The ability to gather continuous, objective data on physical activity and upper limb movements may allow for a more comprehensive understanding of the daily functional capacities of patients with Friedereich's ataxia.

Larimar has undertaken an ongoing open-label extension study of nomlabofusp with the first enrolled patient being dosed in March 2024.

After experiencing setbacks, PTC reported positive results for vatiquinone, its selective inhibitor of 15-Lipoxygenase (15-LO) enzyme, regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia

The new J-code will become effective in January and will provide reimbursement when Exparel is used in the office setting and for office-based surgeries.

Survey finds that people with chronic pain were more supportive of the policies that would expand access to medical cannabis.

A new study highlighted the potential benefits of RSV vaccination, suggesting that the vaccines could provide both individual and societal cost savings.

A review of skeletal muscle relaxants shows that they don't work for pain from osteoarthritis, neuropathic pain and other conditions. Some see an opportunity for more tailored therapeutic strategies, improving patient outcomes.

Most people haven’t heard of the condition, but Friedreich’s ataxia is getting more attention because of a new treatment and a large number of clinical trials enrolling people with the condition.

Results looking at the first season of RSV use shows that it is 75% effective at preventing RSV-associated hospitalizations in those 60 and older.

The future of pain medicine is characterized by treatments and technologies — including virtual reality, AI, wearable tech and psychedelics — that promise to significantly enhance patient care.

Genetic and biochemical testing are crucial for improving outcomes for patients with Friedreich ataxia, according to speakers at a symposium at the European Association of Neurology Congress.

A systemic review of corticosteroid studies finds that their anti-inflammatory effects can be beneficial in addressing peripheral injuries and as a adjuvant for treating the associated pain.