News

May 7, 2021May 7, 2021

Sex, SMN2 Copy Number Influence SMA Type 3 Age of Onset

News

Sex and the number of copies of the “backup” SMN2 gene influence both the age of symptom onset and the risk of losing the ability to walk in people with ... Read more

May 5, 2021May 5, 2021

UK NICE Expands Access to Spinraza for SMA Type 3

News

A decision from England’s National Institute for Health and Care Excellence (NICE) will make Spinraza (nusinersen) available to people with spinal muscular atrophy (SMA) type 3 who are unable to ... Read more

May 3, 2021May 3, 2021

German Study Urges Newborn SMA Screening, Citing Better Outcomes

News

Newborn screening can aid in the early diagnosis and prompt treatment of spinal muscular atrophy (SMA), which can lead to better clinical outcomes, according to a German study. “We strongly ... Read more

April 30, 2021April 30, 2021

Grant Awarded to Find Affordable Treatments for Children

News

A researcher from Keele University School of Medicine in the U.K. was awarded a £99,959 ($138,633) research grant to identify and evaluate potential — and more affordable — treatments to ... Read more

April 28, 2021April 29, 2021

Novartis Poised for Phase 3b SMART Study of Zolgensma

News

Novartis will initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of the one-time gene therapy Zolgensma (onasemnogene abeparvovec) in young children with spinal muscular atrophy ... Read more

April 26, 2021April 27, 2021

Danish Family Races to Raise $2.4M for Daughter’s Zolgensma Therapy

News

Ayah Lundt, a 15-month-old Danish girl with spinal muscular atrophy (SMA) type 2, is helping bring the world together as her parents race to raise $2.4 million for a single dose ... Read more

April 23, 2021April 26, 2021

#AANAM – Zolgensma May Lead to Faster, Greater Gains Than Spinraza

News

Zolgensma may result in faster and greater functional improvements than Spinraza (nusinersen) in infants with spinal muscular atrophy (SMA) treated up to 2 years of age, according to a survey of ... Read more

April 21, 2021April 21, 2021

Quick Approval Given COVID Vaccines Raises Concerns, Rare Disease Patients Say

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More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine ... Read more

April 21, 2021April 21, 2021

#AANAM – Neurofilaments Before Spinraza May Predict Motor Improvements

News

Higher blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before beginning Spinraza (nusinersen) treatment are associated with greater motor improvements in children with ... Read more

April 16, 2021April 16, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

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