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March 1, 2019March 8, 2019

China Approves SMA Treatment Spinraza for Most Patients

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Spinraza (nusinersen) has been approved by China’s National Medical Product Administration (NMPA) for the treatment of the most common form of spinal muscular atrophy (SMA). Marketed by Biogen and ... Read more

February 27, 2019February 25, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have ... Read more

February 27, 2019March 5, 2019

Scientists Create New Method to Calculate Number of SMN1 Copies and Identify SMA Carriers

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Scientists have created a new method to calculate the number of SMN1 gene copies present in individuals and identify possible spinal muscular atrophy (SMA) carriers — those who have only one ... Read more

February 26, 2019February 28, 2019

ICER: Spinraza, Zolgensma Both ‘Dramatically Improve’ Lives of Kids With SMA

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Spinraza (nusinersen), an injectable medication approved two years ago by the U.S. Food and Drug Administration (FDA) to treat spinal muscular atrophy (SMA), is way overpriced. But so is its biggest potential ... Read more

February 25, 2019March 4, 2019

British MP Pushes to Make Spinraza Treatment Available for SMA Patients

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Citing the deteriorating health of a child with spinal muscular atrophy (SMA), a member of the British Parliament is pressing for the treatment Spinraza to be available in England. The ... Read more

February 22, 2019March 7, 2019

New Endurance Tests Can Measure Fatigue in SMA Patients Types 2-4, Study Reports

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Endurance tests that capture upper and lower body fatigue in spinal muscular atrophy patients — across ages, from children to adults, and degrees of disease severity — was proposed ... Read more

February 22, 2019February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The ... Read more

February 20, 2019February 22, 2019

Spinraza’s Cost-Effectiveness Exceeds Swedish Willingness-to-pay Threshold, Study Shows

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Treatment with Spinraza (nusinersen) benefits spinal muscular atrophy patients in terms of survival and quality of life of patients and caregivers, a study conducted in Sweden shows. However, Spinraza treatment ... Read more

February 18, 2019February 22, 2019

RULM Can Assess Arms’ Function in Wider Spectrum of SMA Patients, Study Says

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The Revised Upper Limb Module (RULM) is better than the conventional Upper Limb Module (ULM) at assessing the arms’ function in a wider spectrum of patients with spinal muscular ... Read more

February 15, 2019February 20, 2019

ArcherDX Enters Newborn Screening Market with Baby Genes Acquisition

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ArcherDX is expanding its product portfolio with newborn and screening tests to identify carriers of genetic variants linked to different disorders, including cystic fibrosis, spinal muscular atrophy, and fragile ... Read more

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