Posts by: Patricia Inacio, PhD

News

Optimal care of children with spinal muscular atrophy (SMA) requires improving competence and an adequate exchange of knowledge among healthcare professionals, as well maintaining a close dialogue with the ... Read more

December 21, 2018January 4, 2019

Risdiplam Granted EMA’s PRIME Designation for Potential in Spinal Muscular Atrophy

News

The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of ... Read more

November 14, 2018November 20, 2018

New Technique, MUNIX, May Capture Motor Neuron Loss and Muscle Status in Type 3 and 4 Adults

News

A non-invasive electrophysiology technique called motor unit number index, or MUNIX, can effectively assess the loss of motor nerve cells and muscle impairment in adults with spinal muscular atrophy ... Read more

August 6, 2018August 8, 2018

Mizzou Researchers Launch Shift Pharmaceuticals to Test Potential SMA Therapy

News

Researchers at Mizzou — the University of Missouri — have started a new company, Shift Pharmaceuticals, to test the clinical effectiveness of a molecule that may help improve the ... Read more

July 20, 2018July 24, 2018

Early Spinraza Treatment Improves Outcomes of Children with SMA, Study Shows

News

Young children with spinal muscular atrophy show improvements in motor function after six-months of treatment with Spinraza, a German study shows. The response to Spinraza strongly correlated with the age ... Read more

April 2, 2018April 3, 2018

ALS Research Could Shed Light on What Drives SMA, Study Reports

News

According to scientists, new research on ALS may shed light on the mechanisms underlying spinal muscular atrophy. Two RNA binding proteins, TDP-43 and hnRNP A1, are abnormal in certain cases ... Read more

February 7, 2018February 7, 2018

Genetic Network Identified Regulating Motor Neuron Formation in Embryo’s Development

News

Researchers have uncovered the gene network regulating the transition of progenitor cells into motor neurons during the development of embryos, using chicken and mice as models. The study also ... Read more

February 5, 2018February 5, 2018

Scholar Rock Therapy Prevents Additional Atrophy in Mice with Muscle Wasting, Study Shows

News

Scholar Rock’s SRK-015 prevented additional atrophy in mice with muscle wasting and increased healthy animals’ muscle mass and function, a study reports. The biotech company’s therapy targets the precursor to the ... Read more

January 12, 2018January 12, 2018

Combining Two New Antisense Oligonucleotides Increases SMN Protein Levels in SMA Mice, Study Shows

News

Researchers have shown that the technology called antisense oligonucleotides, used in the only FDA-approved therapy for SMA, Spinraza (nusinersen), can be improved upon. They designed two new sequences that significantly increased SMN ... Read more

December 13, 2017December 14, 2017

Envisagenics Raises $2.35M to Expand Work on RNA-based Therapies for Diseases Like SMA

News

Envisagenics has raised a total of $2.35 million to continue working to discover RNA-based therapies for diseases linked to RNA splicing errors, such as spinal muscular atrophy. The company uses ... Read more

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