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October 22, 2018October 22, 2018

Developers of SMA Therapy Spinraza to Be Honored with $3M Prize at ‘Oscars of Science’

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Pharmacologist C. Frank Bennett, PhD, and biochemist Adrian R. Krainer, PhD, have been awarded a prestigious 2019 Breakthrough Prize in Life Sciences for their role in the development of ... Read more

October 19, 2018October 20, 2018

AVXS-101 May Be ‘Transformative’ Breakthrough for SMA Type 1, Cure SMA President Says

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AveXis‘ gene therapy AVXS-101, now under review for approval to treat infants with spinal muscular atrophy (SMA) type 1 in the U.S. and Europe, may be truly “transformative,” said Kenneth Hobby, president of ... Read more

October 18, 2018October 19, 2018

AveXis Asking FDA, EU to Approve AVXS-101 as 1st Gene Therapy for SMA Type 1

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AveXis has filed a first request for approval of its highly-anticipated gene therapy, AVXS-101, to treat infants with spinal muscular atrophy (SMA) type 1 with the U.S. Food and Drug Administration, CureSMA announced ... Read more

October 17, 2018October 22, 2018

Standard Anesthesia for Spinraza Administration Safe, Effective in Children with Type 2 SMA, Study Shows

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Standard general anesthesia for the administration of Spinraza (nusinersen) is safe and effective in children with type 2 spinal muscular atrophy (SMA), a retrospective study shows. The study, “A ... Read more

October 15, 2018October 19, 2018

Cure SMA Launches Patient Registry, Care Network with Aim of Improving Treatment and Access

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Aiming to establish a standard of care for people affected by spinal muscular atrophy (SMA), Cure SMA has unveiled what’s being touted as the first national clinical data registry ... Read more

October 12, 2018October 17, 2018

Spinraza Retards Disease Progression in Pre-symptomatic SMA Infants, Interim Results of Phase 2 Study Show

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Biogen’s Spinraza (nusinersen) retards disease progression in pre-symptomatic spinal muscular atrophy (SMA) infants, allowing them to achieve motor milestones they would otherwise not reach, according to updated results of ... Read more

October 10, 2018October 18, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 10, 2018October 15, 2018

Risdiplam May Halt Motor Impairment in SMA Patients, Interim Clinical Data Suggests

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The investigational treatment risdiplam (RG7916) improved motor function in patients with type 1, 2 or 3 spinal muscular atrophy (SMA), according to preliminary results from the first part of two ... Read more

October 8, 2018October 15, 2018

Simple Hand Exercise Helps Detect Fatigability in SMA Type 2 Patients, Study Shows

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Patients with spinal muscular atrophy (SMA) are more susceptible to becoming tired during physical activity, which, in addition to muscle weakness, may contribute significantly to their decreased motor function, a study ... Read more

October 5, 2018October 8, 2018

Blood Test for SMN Protein Levels May Help Monitor Treatment Response in SMA, Study Reports

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Researchers in Japan have developed a non-invasive test to measure levels of the SMN protein using cells circulating in the blood of spinal muscular atrophy (SMA) patients. The test is ... Read more

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