Source ALS News Today Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease and Lou Gehrig’s disease, is a rare condition characterized by the degeneration of nerve cells that control voluntary muscles. The disease leads to gradually worsening symptoms that include muscle weakness, twitching, and stiffness. As more of these nerve cells, or motor neurons, are lost,
Two critical reviews examine the current status of biologic approaches for common shoulder and elbow problems. The authors discuss areas where the current evidence base is weak or controversial and recommend where further studies are required. The utilization of stem cell therapies for augmentation of tissue healing has far outpaced the supporting scientific and clinical
Learning how everyday things work in space, such as how to effectively brush teeth or how hair grows, is intriguing, but knowledge of how medical research translates from Earth’s surface to above its atmosphere is limited. A new alliance between the McGowan Institute for Regenerative Medicine at the University of Pittsburgh and the International Space Station (ISS)
Source BioProcess International Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM). Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the
Source: Life Science Leader Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis’ Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the world’s most expensive drug to date. But cell and gene therapies are their own animals; the differences between
Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans. The new variant reduced unintended changes in DNA compared to its wildtype, suggesting it could play a role in gene therapies that require high precision. The study by researchers from Karolinska
The report, sponsored by ARM, Intellia Therapeutics, and CRISPR Therapeutics, and endorsed by former MEP Maria Teresa Giménez Barbat, discusses the commercial, clinical, regulatory, and ethical landscape and framework for the development of therapeutic for genome editing in Europe.
Science is producing a growing number of gene and cell therapies to treat spinal muscular atrophy, leukemia and other conditions, but payers and providers don’t have the information they need to determine what they should pay for these treatments. That’s according to Steven Pearson, MD, founder and president of Boston-based Institute for Clinical and Economic
Instead of augmenting super-soldiers, DARPA wants to boost troops’ natural defenses against engineered diseases — and even undo gene-editing altogether. The Pentagon’s research agency wants to explore the possibility of editing a soldier’s genetic makeup to protect against chemical and biological attacks. Defense Advanced Research Projects Agency director Steven Walker said Monday that he believes
The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds. According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign
