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August 21, 2019August 21, 2019

Delivering Spinraza to Patients with ‘Complex Spines’ Possible Using Different Puncture Route, Study Says

News

An alternative form of intrathecal (via the spinal canal) injection, called a transforaminal puncture, is a safe and effective way of delivering Spinraza (nusinersen) to spinal muscular atrophy patients whose scoliosis makes standard ... Read more

August 19, 2019August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 19, 2019August 20, 2019

BioNews’ Patient Forums Foster Community and Raise Awareness

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Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes ... Read more

August 16, 2019August 20, 2019

Wisconsin Developing Faster, Cost-Effective Method to Screen Newborns for SMA and SCID

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The Wisconsin State Laboratory of Hygiene is using Quantabio‘s technology to improve newborn screening of spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID). The laboratory is developing a faster, easier ... Read more

August 14, 2019August 20, 2019

40 Russian Children Now on Spinraza, as Moscow-based Advocacy Group Pushes for More Treatment

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Doctors have begun administering Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) in Russia for the first time, the head of the country’s SMA patient organization reports. Forty children with the ... Read more

August 12, 2019August 20, 2019

Breathing Tests Could Tell Which Kids Might Need Ventilation, Study Says

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Need for ventilation support is common among children with spinal muscular atrophy (SMA), and those who are older and with more abnormal breathing seem to be at greater risk, ... Read more

August 9, 2019August 20, 2019

Skye, First Baby in Connecticut Treated with Zolgensma, ‘Healthier and Stronger’

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Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is “the happiest child” after being treated with Zolgensma, according to ... Read more

August 8, 2019August 20, 2019

AveXis Knew of ‘Manipulated’ Data in Zolgensma Application and Failed to Inform FDA, Agency Says

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AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation issues” that led to inaccuracies in its application to the ... Read more

August 7, 2019August 7, 2019

’31 Days of SMA’ Among Highlights of August, SMA Awareness Month

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SMA News Today, having launched forums and added regular columnists, is now running a month-long series to coincide with Spinal Muscular Atrophy Awareness (SMA) Month that highlights the challenges and perseverance of patients ... Read more

August 2, 2019August 12, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

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