News

December 18, 2019December 20, 2019

Parents of 2 Babies Treated with Zolgensma in July: We Made the Right Choice

News

The parents of two babies treated in July with Zolgensma, the first gene therapy approved for spinal muscular atrophy or any neuromuscular disease, are confident they made the right choice ... Read more

December 16, 2019December 16, 2019

Lowering EphA4 Gene Expression Has Limited Therapeutic Potential in Mouse Model of Severe SMA, Study Says

News

Lowering the expression of the EphA4 gene — a gene that plays a major role in many neurological disorders — is not enough to improve motor function and survival in ... Read more

December 13, 2019January 8, 2020

Adding Cancer Medicine to Spinraza Boosts SMN Production, Early Study Finds

News

Combining Spinraza with low-dose panobinostat — the active substance in an approved treatment for a blood cancer — may amplify Spinraza’s effectiveness, increasing the production of the survival motor neuron ... Read more

December 11, 2019January 8, 2020

Activities of Daily Living and Caregiver Burden Should be Included in Tools That Measure Quality of Life, Review Says

News

The impact of spinal muscular atrophy on daily life activities and the burden for caregivers should be added to tools currently used in clinical practice to measure health-related quality ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

News

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 9, 2019December 9, 2019

Adults With SMA Tend to Report Low Prevalence of Non-Motor Symptoms, Study Finds

News

Adults with spinal muscular atrophy (SMA) type 2 and 3 tend to report a low prevalence of non-motor symptoms, a study finds. However, some of these symptoms, such as ... Read more

December 6, 2019December 17, 2019

Regular CSF Exams Urged Following Spinraza Treatment to Screen for Injection Side Effects, Study Suggests

News

The work-up of cerebrospinal fluid (CSF) samples from spinal muscular atrophy (SMA) patients treated with Spinraza (nusinersen) revealed mild increases in protein content, which were attributed to lumbar punctures ... Read more

December 5, 2019December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 4, 2019December 4, 2019

Spinraza Stabilizes or Improves Motor Function in Older SMA Patients, US Real-world Study Suggests

News

Spinraza (nusinersen) safely stabilizes or improves motor function in older patients with spinal muscular atrophy (SMA), according to a small U.S. real-world study. Researchers also pointed out that objective measures ... Read more

December 2, 2019December 2, 2019

Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients, US Study Shows

News

Adopting a multidisciplinary approach can optimize pretreatment evaluations and improve the dosing process for Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), a U.S. study shows. This approach, which also used ... Read more

Pin It on Pinterest