Posts by: Ana Pena

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The sense of fatigue perceived by patients with spinal muscular atrophy (SMA) does not correlate with their physical tendency to get tired, their motor function, or quality of life, a ... Read more

November 16, 2018November 20, 2018

LMI070 Oral Candidate For SMA Type 1 Holds Promise, Preclinical Study Shows

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LMI070 (branaplam), a candidate oral therapy under development by Novartis, is being tested in a Phase 1/2 clinical trial for infants with spinal muscular atrophy (SMA) type 1. A recent study ... Read more

November 7, 2018November 13, 2018

3-D Cell Model Mimics SMA Motor Neurons, is Good Platform For Testing Possible Therapies, Study Suggests

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A 3-D cell model derived from patients’ stem cells is able to mimic motor neurons from spinal muscular atrophy (SMA) patients and can become a better physiological tool to ... Read more

October 8, 2018November 16, 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

August 27, 2018September 10, 2018

Phase 2 Trial Evaluating Mestinon, Already Approved for Myasthenia Gravis, for SMA Types 2-4

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A Phase 2 clinical trial is testing the effectiveness of Mestinon (pyridostigmine) — an approved medicine for another neuromuscular disease — in treating fatigue in people with spinal muscular atrophy ... Read more

August 20, 2018September 10, 2018

Psychological Support Relieves Anxiety of Spinraza Treatment in Kids with SMA Type 1, Study Says

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Psychological interventions to control emotional stress can help relieve the anxiety that parents and children with spinal muscular atrophy type 1 feel during Spinraza administration through spinal taps, an ... Read more

August 3, 2018August 10, 2018

New Mutation Linked to SMA-LED in 4 Unrelated Children, Case Study Reports

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A newly identified mutation in the DYNC1H1 gene causes a rare form of spinal muscular atrophy known as SMA with lower limb predominance, which is associated with learning difficulties and brain ... Read more

July 18, 2018August 10, 2018

Case Report Alerts Physicians About Link Between SMA and Ketoacidosis

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Spinal muscular atrophy (SMA) patients can be more prone to a life-threatening metabolic imbalance called ketoacidosis, which causes the blood to become acidic, a case report highlights. In patients with ... Read more

July 13, 2018July 17, 2018

Protein Independent of SMN May Be Worthy Target as Combo Treatment for SMA, Mouse Study Suggests

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Lowering the levels of a protein called CHP1, together with Spinraza (nusinersen) treatment, may help to improve responses to therapy in spinal muscular atrophy (SMA) patients, a study in a mouse model of the ... Read more

July 9, 2018July 12, 2018

New Potential Therapeutic Target for SMA Identified in Mouse Study

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A key player in a pathway known as the cell’s “cleaning system” could be a promising target for alternative approaches to treating spinal muscular atrophy (SMA), a mouse study suggests. The ... Read more

Posts by: Ana Pena

Ana Pena

Perceived Fatigue Not Linked to Physical Factors, Quality of Life in SMA Patients, Study Reports

LMI070 Oral Candidate For SMA Type 1 Holds Promise, Preclinical Study Shows

3-D Cell Model Mimics SMA Motor Neurons, is Good Platform For Testing Possible Therapies, Study Suggests

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

Phase 2 Trial Evaluating Mestinon, Already Approved for Myasthenia Gravis, for SMA Types 2-4

New Mutation Linked to SMA-LED in 4 Unrelated Children, Case Study Reports

Case Report Alerts Physicians About Link Between SMA and Ketoacidosis

Protein Independent of SMN May Be Worthy Target as Combo Treatment for SMA, Mouse Study Suggests

New Potential Therapeutic Target for SMA Identified in Mouse Study

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