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In a response to a survey, caregivers of people with spinal muscular atrophy identified the risk of severe adverse events and the need for permanent ventilation as the most important factors in treatment decisions. Access to treatment, including cost and availability, ranked third.

Mouse models of liver, kidney and lung fibrosis showed that genetic deletion of MERTK prevented fibrosis

If approved, donidalorsen would be a first-in-class RNA-targeted medicine for hereditary angioedema. The agency’s goal date is August 21, 2025.

Here’s what you missed this week on Managed Healthcare Executive.

Type 1 diabetes is a relentless disease, according to David Maahs, M.D., M.A., Ph.D., from Stanford University.

The rising popularity of the continuous glucose monitor, which is not the same as an insulin pump, has been a huge advance in type 1 diabetes treatment, according to David Maahs, M.D., M.A., Ph.D., from Stanford University.

David Maahs, M.D., M.A., Ph.D., explains why teamwork is the most important part of the 4T diabetes management program.

David Maahs, M.D., M.A., Ph.D., explains the role that teamwork, targets, technology and tight control plays in type 1 diabetes management and why it’s best to intervene right after diagnosis.

Scemblix (asciminib) is a new first-line option for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).

Artificial intelligence stands out as a particularly promising solution to the challenges of improving health literacy.

Prademagene zamikeracel (pz-cel) could be a new treatment for rare genetic disease recessive dystrophic epidermolysis bullosa (RDEB).

A single-center retrospective study found the approval and use of new agents to treat chronic graft-versus-host disease (GVHD) is associated with quality-of-life improvements.

Here’s what you missed this week on Managed Healthcare Executive.

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

Popular menopause prescriptions such as Estradiol, Dotti, Premarin and Veozah will be available for cheaper using a GoodRx coupon.

A panel of speakers demonstrated different ways technology and artificial intelligence (AI) could greatly expand access to glaucoma care.

This week’s approval expands Seldardsi indications to treat adults with Crohn’s disease and ulcerative colitis.

Changes have been proposed to the quality payment program that could have ups and downs, but not, perhaps for some, until 2029.

A new statistical models shed light on why some patients can extend the dosing interval with Eylea (aflibercept) from 8 weeks to as long as 20 weeks for neovascular age-related macular degeneration (nAMD) or diabetic macular edema (DME).

Stephanie Graff, M.D., FACP, FASCO, director of breast oncology at the Lifespan Cancer Institute and author of Investigating the Salience of Clinical Meaningfulness and Clinically Meaningful Outcomes in Metastatic Breast Cancer Care Delivery, shares the reasons why she chose to study metastatic breast cancer patients.

AI (artificial intelligence) can improve the accuracy of disease prediction by crunching volumes of existing clinical data on patients.

There is a need for strict policies regarding the use of AI in the managed care space, according to Douglas S. Burgoyne, Pharm.D., MBA, FAMCP, adjunct associate professor at the University of Utah College of Pharmacy.

Health plans and PBMs are using AI to aggregate data, summarize information and enhance their formulary decision-making processes, according to Douglas S. Burgoyne, Pharm.D., MBA, FAMCP, adjunct associate professor at the University of Utah College of Pharmacy.

Technology can help address the complexities of specialty medications by automating processes and enabling proactive interventions to ensure patients stay on their critical therapies, according to Kristen Whelchel, Pharm.D., CSP, Research and Patient Care Improvement Pharmacist with Vanderbilt Specialty Pharmacy's Department of Health Outcomes and Research.

Up to half of patients showed significant improvement in study results presented at the American Academy of Ophthalmology (AAO) annual meeting

Here’s what you missed this week on Managed Healthcare Executive.

In the threat versus opportunity session, Cecilia and Aaron Lee of the University of Washington staked out opposing positions.

Patients self-identified as members of the minority group that the study was designed to enroll, according to Jeremiah Brown, M.D., of Retina Consultants of Texas, one of the lead ELEVATUM investigators. The study will yield a "treasure trove" of data, Brown said.

Enrollment goals for the U.S. part of the phase 4, open-label study of Vabysmo (faricimab) were 45% Black participants, 45% Hispanic participants and 10% Native American, Pacific Islander and Native Hawaiian, said Jeremiah Brown, M.D., a lead ELEVATUM investigator.

Harmony Garges, M.D, senior vice president, chief medical officer and head of global medical at ViiV Healthcare, highlighted results of two real-world evidence studies of Apretude as an HIV prevention medication.