Posts by: Magdalena Kegel

Magdalena Kegel

Magdalena is a writer with a passion for bridging the gap between the people performing research, and those who want or need to understand it. She writes about medical science and drug discovery. She holds an MS in Pharmaceutical Bioscience and a PhD — spanning the fields of psychiatry, immunology, and neuropharmacology — from Karolinska Institutet in Sweden.

January 22, 2018January 22, 2018

Arm Cycling Increases SMA Patients’ Endurance, but Not Levels of SMN Protein, Study Finds

Arm cycling training increased spinal muscular atrophy type 2 patients’ endurance and improved their results on movement tests, a small pilot study indicated. Despite researchers’ expectations, the training had ... Read more

January 19, 2018January 19, 2018

AveXis Expands Clinical Testing of Gene Therapy, AVXS-101, to Include Milder SMA Types

AveXis plans to launch clinical trials of its gene therapy AVXS-101 in a wider range of spinal muscular atrophy (SMA) patients — mirroring the company’s belief that the treatment ... Read more

January 8, 2018January 8, 2018

FDA Lets AveXis Know the Information It Needs to Possibly Approve AVXS-101 for SMA Type 1

The U.S. Food and Drug Administration has given AveXis a list of information it needs to consider approving AVXS-101 as a treatment for spinal muscular atrophy Type 1. Company officials met with ... Read more

December 20, 2017January 2, 2018

Spinal Muscular Atrophy Researchers Identify Spinraza Ethical Challenges

Although Spinraza has the potential to change the course of spinal muscular atrophy, a number of ethical challenges revolve around its use, a group of American researchers argues. In an article ... Read more

December 18, 2017December 18, 2017

Faulty Molecular Transport Process May Lead to Movement Nerve Cell Death, Study Reports

A faulty molecular transport process could explain how loss of a protein leads to the death of movement nerve cells that is seen in spinal muscular atrophy, a study reports. ... Read more

December 15, 2017

FDA Gives AveXis Thumbs Up to Launch Gene Therapy Study in SMA Type 2

The U.S. Food and Drug Administration (FDA) has given AveXis the green light to launch a Phase 1 study of its gene therapy, AVXS-101, in children with spinal muscular atrophy ... Read more

December 4, 2017December 5, 2017

Cytokinetics’ New Muscle Activator May Have Better Tolerability, Effectiveness Than Tarasemtiv

Cytokinetics’ new muscle activator compound shows promising safety, tolerability, and effectiveness in three early clinical trials in healthy volunteers. The data supports the ongoing Phase 2 trial of the ... Read more

December 1, 2017

SMA Type 1 Study of Disease’s Natural Course Supports Use of Newborn Screening

A study of the natural course of spinal muscular atrophy (SMA) type 1 supports the idea that treatment should be attempted as early as possible — further supporting arguments ... Read more

November 29, 2017November 30, 2017

People in the UK Support Newborn SMA Screening, Study Shows

The majority of people that completed a U.K. survey support the idea of newborn screening for spinal muscular atrophy , according to a report published in the journal Molecular ... Read more

November 22, 2017

Catalyst Launches Proof-of-Concept Trial of Firdapse for SMA 3 Symptom Relief

Catalyst Pharmaceuticals is launching a proof-of-concept Phase 2 trial of its investigational drug Firdapse (amifampridine phosphate), intended to relieve symptoms in ambulatory patients with spinal muscular atrophy (SMA) type ... Read more

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