
Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.

Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.

Disease-modifying agents will require early detection, including comprehensive evaluation of disease severity and alternative or confounding diagnoses. When the long-awaited moment arrives, when disease-modifying therapies finally are available for ADRDs…will we be ready?

The holy grail of neurodegenerative disorders would be to alter or halt the progression of the disease. For years, the discussion has swirled around stem cells and gene therapy, and they remain potential options for the future.

With an anticipated life expectancy of 3 years from the time of symptom onset, an effective treatment strategy is essential in ALS—and recent therapeutic progress has built a foundation of hope for the community.

Disease pathogenesis is attributed to oxidative stress—which can be regulated by NRF2, which, in turn, binds to antioxidant responsive elements in the promoter of the target gene FXN to control its expression.

Several therapies are progressing through the development pipeline and have shown promising data, setting up the multiple sclerosis treatment toolbox for possible expansion in coming years.

Since the days of limited treatment in neurology, 30 years of progress have brought an expanded armamentarium of therapies for many neurological disorders.

One model of AD suggests that Aβ pathophysiology triggers downstream molecular pathways, including tauopathy, which lead to cortical neurodegeneration, and cognitive decline is further attributed to the associated neurocortical Aβ plaques.

Whether it is a standard of care that is practical but not often appreciated or an emerging treatment that can change the landscape of a devastating disease, neurology as a whole is fueled by hope, and that is a gift that keeps on giving.

Cognitive problems have been reported to occur in as many as 7 in 10 individuals with MS, yet they remain a difficult and undertreated challenge in clinical care.

The paradigm-shifting evidence of the link between MS and EBV does not exist in isolation. In fact, the relationship has been suspected for more than 40 years, and evidence therein has been accumulating over the past 2 decades.

Fatigue, although not immediately visible, is among the most debilitating symptoms of multiple sclerosis, affecting not only patients' day-to-day functioning but their quality of life.

Cognitive and other symptoms in multiple sclerosis can be invisible—meaning not readily observed in the individual. However, their impact on a patient’s life can be obvious.

Available neurodegenerative disease treatments are generally unsafe and ineffective at penetrating the blood-brain barrier, though the use of nanoparticles can provide improved penetration and exert a neuroprotective effect.

The Palatucci Advocacy Leadership Forum, or PALF, sponsored by the American Academy of Neurology, gives neurologists and trainees tools to successfully advocate for their ideas and develop their identity as physician advocates.

Acting as an advocate is meaningful to neurologists because it speaks to their inner core—or their “why.” Neurologists have the agency to make this world a better place while working as a neurologist and an advocate at many levels.

The long-term consequences of untreated—and prevalent—sleep disorders in children and adolescents point to a need for a focus on this field of care.

Identified implications of the endocannabinoid system in migraine physiology suggest that this pathway might hold therapeutic potential for some headache disorders.

Neuromuscular medicine has entered an era of genetic therapy, opening the doors to questions about possible combination approaches and earlier initiation of treatment.

Despite the availability of medications to relieve OFF episodes in Parkinson disease, widespread adoption of on-demand therapies has been less-than-ideal, marking the need to shift the treatment paradigm in PD.

Progress in therapeutics and diagnosis over the past decade has dramatically shifted the treatment landscape for this genetic epilepsy and rapidly advanced patient care.

Achieving greater diversity in study populations is a focus of increasing scrutiny, priority, and funding in all medical research, including studies enrolling individuals on the continuum of prodromal Alzheimer disease, mild cognitive impairment, and Alzheimer disease.

Although new treatments and innovations are a sign of growth in migraine care, it is important to realize that growth comes with a certain amount of pain. In clinical practice, choosing the right treatment option for patients in a time-limited visit is often a dilemma.

Given the importance of mitochondrial and endoplasmic reticulum function in ALS, the disruption of intracellular mitochondria-endoplasmic reticulum contacts presents yet another avenue for neuronal degeneration—perhaps the primary point of underlying dysfunction.

The journey of aducanumab (Aduhelm; Biogen) from development to the FDA is a tortured one, but its path may serve to teach the Alzheimer disease field valuable lessons as it strives forward to develop disease-modifying therapies.

The pandemic has highlighted several large gaps in care that have created challenges for individuals with Parkinson disease, including a framework for addressing mental health and loneliness, and proper care for women with PD.

While shutdowns disrupted care for all patients with Parkinson disease, hospitals were forced to postpone elective procedures such as the initial DBS device implantation and implantable pulse generator replacements.

Despite progress in the understanding of Tourette syndrome and similar disorders, no clear cause of TS has been identified, nor are there treatment options that completely eliminate symptoms.

Parkinson disease is neither imminently fatal nor transient but is incurable, and as it affects individuals differently based on their unique identities, culture, access to health care, and social support, it is vital to empower patients.

With the physician deficit projected to grow larger within a decade, this global challenge has become a major focus of large organizations and medical societies.