April 2022

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.

The study evaluating 68 boys and young men with Duchenne muscular dystrophy will consist of therapy with off-the-shelf cardiosphere-derived cells.

David Kudrow, MD, provides expert insight on the newest options for migraine management and prevention.

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.

After being originally approved as a generic medication in 1996, a new transdermal formulation of donepezil is expected to be available for patients with Alzheimer disease in the fall of 2022.

Individuals on ravulizumab demonstrated statistically significant changes in the primary end point of MG-ADL and in secondary end points such as Quantitative Myasthenia Gravis total score.

Data from the phase 2 NURTURE study of nusinersen (Spinraza; Biogen) suggest that long-term treatment with the agent is beneficial, and point to the importance of newborn screening and early treatment for spinal muscular atrophy.

A third dose enhanced the number of responders to all variants and significantly increased CD8 T-cell responses, but the frequencies of Omicron-specific CD8 T-cells remained 70% of the responses specific to the vaccine strain.

The trial will use PACC-5 score as its primary end point and expects to enroll 1200 participants who have confirmed amyloid pathology without signs of cognitive impairment.

The treatment is being developed as a first-in-class regenerative therapy for those with AD and dementia, with these safety data offering support to its development.

The two investigational agents from 4D Pharma have both been shown in preclinical studies to reduce neuroinflammation and to protect neurons from oxidative stress-induced death.

Mechanistic data from the phase 2 REPAIR-MS trial support blinded efficacy data from the VISIONARY-MS trial, suggesting that Clene Nanomedicine’s investigational CNM-Au8 has the potential to drive clinically meaningful improvements in recognized MS functional end points.

For patients who remained on 60-mg tolebrutinib through Part B of the study, the number of new Gadolinium-enhancing lesions and new/enlarging T2 lesions remained low.