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Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)

Share this article   SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2 2,3 Zolgensma (onasemnogene abeparvovec) is approved for the treatment of pediatric SMA patients with 3 or fewer copies of the SMN2 gene, or infantile-onset SMA 1 Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression 1 DORVAL, QC, Dec. 16, 2020 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that Health Canada has approved Zolgensma ® (onasemnogene abeparvovec) an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (

COVID-19 vaccine trials report cases of brief facial paralysis That s not as scary as it sounds

COVID-19 vaccine trials report cases of brief facial paralysis. That s not as scary as it sounds. Adrianna Rodriguez, USA TODAY Replay Video UP NEXT Americans are increasingly concerned about vaccine safety after four people in Pfizer-BioNTech trials and three people in the Moderna trials developed Bell’s palsy, a condition that causes temporary weakness or paralysis of the facial muscles. While it may sound scary, experts say Bell’s palsy is more common and less severe than people think. Bell’s palsy, also known as peripheral facial nerve palsy, can occur at any age, according to the Mayo Clinic. The exact causes are unknown, but it’s believed to be the result of swelling and inflammation of the nerve that controls the muscles on one side of the face, or a reaction after a viral infection.

InnoSkel Launches with €20 Million Series A Financing

December 14, 2020 InnoSkel Launches with €20 Million Series A Financing GlobeNewswire InnoSkel Launches with €20 Million Series A Financing Proceeds to support development and advancement of innovative gene therapies for patients with rare skeletal diseases Founded and led by industry veteran and renowned scientist, Elvire Gouze, PhD Series A Financing co-led by Jeito Capital and Vida Ventures, with support from Turenne Group and Région Sud Investissement Nice, France, 14 December 2020 – InnoSkel, a newly launched platform biotechnology company pioneering gene therapies for rare skeletal diseases, today announced that it has raised €20 million in a Series A financing round. The fundraise was co-led by Jeito Capital and Vida Ventures, with additional support from Turenne Group and Région Sud Investissement. Proceeds from the financing will be used to advance the company’s lead program to clinical stage operations. InnoSkel was founded by Elvire Gouze, PhD, a scien

Is It a Migraine Attack or a Cluster Headache?

Medically Reviewed Both migraine and cluster headache tend to affect primarily one side of the head, but cluster headache typically causes a runny nose and tearing eye in addition to pain. iStock; Everyday Health Whether you’re having a migraine attack or a cluster headache, the pain it causes can be debilitating. But because the treatments for the two diseases are very different, it’s crucial to figure out which you have or whether you have something else entirely to have the best chance at easing your pain. Without the correct diagnosis, it’s difficult to find relief for your headache, says Merle L. Diamond, MD, associate director of the Diamond Headache Clinic in Chicago.

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