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Quantitative magnetic resonance imaging seemed to show that delandistrogene moxeparvovec protects muscle from progressive damage in DMD. | Image credit: OlegKachura - stock.adobe.com

May 14th 2025

Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.

While previous research on the DVA focused on task-specific measurements, the new study focused on total composite severity percentage among patients with DMD. | Image credit: ChiccoDodiFC - stock.adobe.com

April 18th 2025

At-Home DMD Video Assessment Found Valid, Reliable

Rose McNulty

The remote measurement tool enables Duchenne muscular dystrophy (DMD) assessments through analysis of caregiver-recorded videos.

Kavita Nair Q&A - Maks_Lab - stock.adobe.com.jpeg

March 12th 2025

Coming Together to Ensure Neurological Treatment Access: Q&A With Kavita V. Nair, PhD

Christina Mattina Kavita V. Nair, PhD

In 2025, each issue of Population Health, Equity & Outcomes will feature a profile of a health system leader transforming care in their area of expertise. This issue spotlights a conversation with Kavita V. Nair, PhD, of the University of Colorado Anschutz Medical Campus.

February 26th 2025

Adaptive Deep Brain Stimulation Technology Receives FDA Approval for Parkinson Disease

Giuliana Grossi

The FDA approves BrainSense Adaptive deep brain stimulation and electrode identifier technology for the treatment of Parkinson disease.

$Vertebral fractures are common in glucocorticoid-associated osteoporosis and are a clinical indicator of osteoporosis in young patients. | Image credit: Hugnaka - stock.adobe.com$

February 14th 2025

Adults With DMD More Than Twice as Likely to Experience Fractures

Rose McNulty

Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of osteoporosis in adult men with Duchenne muscular dystrophy (DMD).

Parkinson disease | Image credit: wladimir1804 - stock.adobe.com

February 4th 2025

FDA Approves First Subcutaneous Apomorphine Device for Parkinson Disease

Skylar Jeremias

The FDA has approved the first and only subcutaneous apomorphine infusion device, apomorphine hydrochloride injection (Onapgo), for the treatment of adults with advanced Parkinson disease.

Stroke. | Image Credit: Chinnapong - stock.adobe.com

January 30th 2025

New Model Predicts Stroke Severity and Improves Functional Independence

Cameron Santoro

A new predictive model developed by Ochsner Health helps clinicians make real-time decisions for patients who had a stroke undergoing urgent carotid intervention, improving chances of regaining functional independence.

Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com

January 28th 2025

EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD

Rose McNulty

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).

FDA headquarters | Image Credit: Grandbrothers - stock.adobe.com

January 27th 2025

FDA Approves New Maintenance Dosing for Lecanemab to Treat Early-Stage Alzheimer Disease

Brooke McCormick

The FDA approves a new maintenance dosing regimen for lecanemab (Leqembi; Eisai) in patients with early-stage Alzheimer disease (AD), enabling a transition from biweekly to once-every-4-week dosing while preserving clinical and biomarker benefits.

Heart and stethoscope | Image credit: inthasone - stock.adobe.com

January 24th 2025

Prophylactic Cardiac Medication May Prolong Survival in DMD

Rose McNulty

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found.

Past studies have explored the association between motor and cardiac function in younger patients with DMD, but there is a lack of research on this relationship as patients with DMD age. | Image credit: Nana_studio - stock.adobe.com

January 13th 2025

Later Loss of Ambulation Linked With Better Heart Health in DMD

Rose McNulty

The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).

Patients with MS had higher odds of filling prescriptions for anticonvulsants and higher odds of filling gabapentinoids and gabapentinoids without other anticonvulsants in the years leading up to onset of MS. | Image credit: Vitalii Vodolazskyi - stock.adobe.com

January 6th 2025

Pain May Be Indicator of MS Prior to Disease Onset: Study

AJMC Contributor

Compared with healthy controls, patients with multiple sclerosis (MS) had higher odds of filling prescriptions for gabapentinoids and other anticonvulsants in the 5 years prior to MS onset.

Better understanding the vascular sequelae of MS may similarly unlock predictive insights for people with MS, according to investigators. | Image credit: Elena - stock.adobe.com

January 3rd 2025

Meta-Analysis: Vascular Function Worse in People With MS

Jared Kaltwasser

The differences did not appear to be associated with potential moderating factors like age, sex, or smoking status.

lab test tubes | Image credit: kwanchaift – stock.adobe.com

December 22nd 2024

Study Highlights Trends, Emerging Innovations in DMD Research

Hayden E. Klein

An analysis revealed global trends and emerging focus areas in Duchenne muscular dystrophy (DMD) research, emphasizing the growing impact of precision medicine and gene therapies.

Nurse helping patient use walker | Image credit: LIGHTFIELD STUDIOS – stock.adobe.com

October 9th 2024

Genotypes May Influence When Patients With DMD Have Loss of Ambulation

Hayden E. Klein

A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).

Currently, 4 exon-skipping drugs are FDA approved for DMD: eteplirsen (Exondys 51), golodirsen (Vyondys 53), casimersen (Amondys 45), and viltolarsen (Viltepso) | Image credit: CrazyJuke - stock.adobe.com

September 26th 2024

WVE-N531 Shows Promising Efficacy in DMD at Interim FORWARD-53 Analysis

Rose McNulty

In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

The new results make HERCULES the first and only study to demonstrate reduced CDP in nrSPMS. | Image credit: Juan Gärtner - stock-adobe.com

September 3rd 2024

Tolebrutinib Delays Disability Progression in Nonrelapsing Secondary Progressive MS

Rose McNulty

The HERCULES study of tolebrutinib is the first and only to show reduced confirmed disability progression at 6 months in nonrelapsing secondary progressive multiple sclerosis (MS).

Balance and gait are both important factors in continued mobility among patients with DMD. | Image credit: OlegKachura - stock.adobe.com

August 22nd 2024

Cycling Training Shows Protective Effects on Gait, Balance in DMD

Rose McNulty

Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior balance.

Results demonstrate correlations between fatigue and walking speed and mobility among patients with multiple sclerosis. | Image credit: kieferpix - stock.adobe.com

Published: August 13th 2024 | Updated: August 14th 2024

Fatigue Linked With Reduced Walking Speed, Mobility in Multiple Sclerosis

Rose McNulty

Study results demonstrated significant correlations between fatigue and reductions in walking speed and mobility among patients with multiple sclerosis.

Healthy behaviors, especially regular exercise and a healthy diet, were associated with better patient-reported outcomes in multiple sclerosis. | Image credit: Daniel Vincek - stock.adobe.com

August 7th 2024

Healthy Lifestyle Behaviors Linked to Better MS Outcomes

Rose McNulty

Engaging with healthy lifestyle behaviors, including a healthy diet and physical activity, was associated with better outcomes among patients with multiple sclerosis (MS).

A panel of 5 experts on Alzheimer disease

June 26th 2024

Payer Coverage Considerations for New Treatments for Alzheimer Disease

Derek van Amerongen, MD, MS Soeren Mattke, MD, DSc B. Joy Snider, MD, PhD Kavita V. Nair, PhD Maria Lopes, MD, MS

Experts discuss how payers can prepare for new treatments and plan adequate coverage for patients with Alzheimer disease.

Blue stamp of FDA approval | Image Credit: argus-stock.adobe.com

June 20th 2024

FDA Grants 2 Approvals to Delandistrogene Moxeparvovec for DMD

Maggie L. Shaw

Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.

June 19th 2024

Exploring the Influence of Administration Routes on Medical Costs and Caregiver Burden

Derek van Amerongen, MD, MS Soeren Mattke, MD, DSc B. Joy Snider, MD, PhD Kavita V. Nair, PhD Maria Lopes, MD, MS

An overview of how disease-modifying treatments for Alzheimer disease can impact medical costs and caregiver burden.

June 19th 2024

Advancements in Alzheimer Disease Treatment: Emerging Drug Candidates

Derek van Amerongen, MD, MS Soeren Mattke, MD, DSc B. Joy Snider, MD, PhD Kavita V. Nair, PhD Maria Lopes, MD, MS

The panel discusses emerging therapies for Alzheimer disease that are currently in development, including donanemab, subcutaneous lecanemab, and ALZ-801.

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