Neurology

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.

Explore 5 rare genetic and neurological disorders named after pioneering women in medicine, highlighting their significant contributions to previously unknown conditions.

The FDA cleared marketing for the first in vitro diagnostic device that tests blood to aid in diagnosing Alzheimer disease.

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.

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

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for patients with Duchenne muscular dystrophy (DMD).

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.

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

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).

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.

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.

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

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.

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

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

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.

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

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.

Sub Subramony, M.D., discusses strategies to enhance support for patients with Friedreich ataxia in clinical settings and the broader community, along with his aspirations for future research aimed at addressing unmet needs in treatment and patient support.

Sub Subramony, M.D., discusses the most prevalent complications and comorbidities in Friedreich ataxia, emphasizing the frequent cardiac complications and their significant impact on mortality rates.

Sub Subramony, M.D., discusses the challenges faced by patients with Friedreich ataxia in accessing healthcare services and managing their condition, as well as the significant burden the disease places on families and caregivers.

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

Sub Subramony, M.D., discusses how the progression of patients' conditions often leads to a decline in quality of life and functional capabilities, highlighting key clinical indicators that necessitate the adoption of mobility aids.

Sub Subramony, M.D., discusses how the modified Friedreich Ataxia Rating Scale (mFARS) is valuable for monitoring disease progression in clinical trials and its applicability in everyday clinical practice for assessing patient status.

Panelists discuss how the differential diagnosis for Friedreich's Ataxia involves distinguishing it from other ataxias, the process of definitive diagnosis through genetic testing, and the significant emotional and practical impact that such a diagnosis has on affected families.