Cure SMA

The manuscript was published in the May 2021 issues of BMC Pediatrics.

A pharmacokinetic/pharmacodynamic model indicated that the exposures predicted with a higher dose of nusinersen may lead to more clinically meaningful increases of at least 5 points on CHOP INTEND score.

Two experts in spinal muscular atrophy (SMA) care, Diana Castro, MD, and Garey H. Noritz, MD, offer tips and demonstrations for screening and diagnosing SMA via telehealth, and they outline resources provided by Cure SMA for families and clinicians who suspect, or have a patient with a diagnosis of, SMA.

A recent publication describes the findings of two initiatives to evaluate and raise awareness about the impact that SMA has on adolescents and young adults.

The Adults with SMA Virtual Therapy Program is sponsored by Genentech and the Dhont Family Foundation.

Closing thoughts on advice to community neurologists treating spinal muscular atrophy (SMA).

Experts remind families of the importance of follow-up visits with the multidisciplinary team during COVID-19.

Resources are discussed, including the Child Neurology Foundation’s telehealth tip sheet, to help families navigate telehealth.

Several spinal muscular atrophy (SMA) resources are suggested to help physicians and families, including Cure SMA and standard of care guidelines.

Discussing the diagnosis of spinal muscular atrophy (SMA) with the family and lining up appointments with the multidisciplinary team to help care for the patient.

Diana Castro, M.D., stresses the value of the initial in-person neurology visit for evaluation of spinal muscular atrophy (SMA) to ensure a correct diagnosis. The next step in the process would be genetic testing.

The signs and symptoms leading a pediatrician to a diagnosis of spinal muscular atrophy (SMA) are discussed.

Diana Castro, M.D., discusses newborn screening for spinal muscular atrophy (SMA) and suggests how to care for patients after the results are received.

Early recognition and referral of a patient with suspected spinal muscular atrophy (SMA) to a neurologist.

Garey H. Noritz, MD, walks us through a virtual pediatric neurological exam and notes examination findings that may be identified in an infant with Spinal Muscular Atrophy (SMA).

Diana Castro, MD walks us through a pediatric neurological exam and notes examination findings that may be identified in an infant with Spinal Muscular Atrophy (SMA).

The director of research analytics at Cure SMA detailed the ways to decrease care management costs for patients with spinal muscular atrophy and the role incoming treatments can play.

The director of research analytics at Cure SMA discussed her research on the economic burden and costs patients with spinal muscular atrophy and their caregivers face.

Lisa Belter, MPH, director of research analytics at Cure SMA, discussed a recently published study on the rising costs of living for patients with spinal muscular atrophy.

The team Cure SMA endurance program goes virtual - sign up now.

The average number of inpatient claims per year was higher for patients with SMA than their matched non-SMA patients, with respiratory illness the most common reason for an inpatient admission among all patients with SMA.

The webinars, for adults and children with SMA, were produced in conjunction with Biogen and the Luke 18:1 Foundation.

The 3-part educational program is sponsored by Genentech.

The chief medical officer at Cure SMA spoke to the ongoing work that the organization does in advocating for patients with spinal muscular atrophy, particularly for newborn screening.

The chief medical officer at Cure SMA detailed the importance of having options for patients and how certain administration routes and schedules offer more personalized approaches, particularly for adult patients with SMA.

The chief medical officer at Cure SMA detailed the importance of having various treatment options for patients, particularly for adults with SMA.

The chief medical officer at Cure SMA offers insight on the current SMA therapeutic landscape and what the organization is focused on going forward.

Advocacy organization Cure SMA provides guidance for health care providers caring for patients with SMA during the ongoing COVID-19 pandemic.

Cure SMA gives an overview of the recent 2020 Virtual SMA Conference, with highlights on new data and poster sessions.

The latest program from Cure SMA aims to better understand the capacity of spinal muscular atrophy centers in the US and to continue to carry out effective clinical trials.