Muscular Atrophy News and Research

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FDA approves first drug to treat children and adults with spinal muscular atrophy

The U.S. Food and Drug Administration today approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.

23 Dec 2016

Advances in RNA research could pave new way for development of anti-cancer drugs

An international research team led by the University of Leicester has made a breakthrough advance that could pave a new route for the development of anti-cancer drugs.

8 Nov 2016

Research findings point to potential new targeted therapy for SMA

For the first time, scientists found that in spinal muscular atrophy (SMA), the affected nerve cells that control muscle movement, or motor neurons, have defects in their mitochondria, which generate energy used by the cell.

15 Sep 2016

Scientists find new target that may lead to future treatments for Kennedy's disease

If a disease affects motoneurons, cells that control voluntary muscle activity, researchers should focus their efforts on motoneurons to find potential treatments, right? Not always.

31 Aug 2016

HMS study reveals key instigator of nerve cell damage in ALS patients

Scientists from Harvard Medical School have identified a key instigator of nerve cell damage in people with amyotrophic lateral sclerosis, or ALS, a progressive and incurable neurodegenerative disorder.

25 Aug 2016

First investigational treatment for infantile-onset SMA shows promising results in clinical trial

A major milestone was reached when nusinersen, an investigational treatment for spinal muscular atrophy (SMA), was shown to significantly improve achievement of motor milestones in babies with infantile-onset SMA, according to an interim analysis of the double-blind, randomized, placebo controlled Phase 3 clinical trial called ENDEAR.

11 Aug 2016

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.

25 Jul 2016

Using NMR to investigate intrinsically disordered proteins: an interview with Dr Isabella Felli

“IDPs” is now a widely used acronym that stands for “intrinsically disordered proteins.” It is the term generally used by the scientific community to refer to a wide variety of proteins that do not have a stable 3D structure and are instead characterized by a high extent of local mobility, disorder and many conformers that are accessible at room temperature.

From Bruker BioSpin Group 30 Jun 2016

New paediatric exoskeleton aims to support children with spinal muscular atrophy

Furthermore, it will also be used in physiotherapy in hospitals to prevent the secondary effects associated with the loss of mobility in this illness. The technology, which has been patented and licensed jointly by CSIC and its technology-based business unit, Marsi Bionics, is currently in the preclinical phase.

9 Jun 2016

Bereaved parents feel that pediatric end-of-life care needs improvement

Many pediatricians and pediatric subspecialists believe that their clinical care extends from treating ill children through end-of-life care. However, are pediatricians actually meeting the needs of families and their dying child? In a new study scheduled for publication in The Journal of Pediatrics, researchers surveyed bereaved parents and found that pediatric end-of-life care needs improvement.

27 May 2016

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Michigan State University researchers used an old-fashioned neurobiology technique to explore new avenues for treatments to reverse a late-onset neurodegenerative disease that robs men of the capacity to walk, run, chew and swallow.

9 May 2016

Gene-replacement therapy could be potential treatment option for SMARD1

Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death.

6 May 2016

Long-term exercise may benefit Spinal Muscular Atrophy patients

Long-term exercise appears to be beneficial for Spinal Muscular Atrophy (SMA) like mice, suggesting a potential of active physiotherapy for patient care; according to a study published today in the Journal of Physiology.

6 Mar 2016

Dr. Elliot J. Androphy receives IGNITE grant to develop drug candidate for spinal muscular atrophy

Dr. Elliot J. Androphy, the Kampen-Norins Professor and chair of the Department of Dermatology at the Indiana University School of Medicine, has been awarded an IGNITE, or Innovation Grants to Nurture Initial Translational Efforts, grant by the National Institutes of Health.

22 Feb 2016

Muscular Atrophy News and Research

FDA approves first drug to treat children and adults with spinal muscular atrophy

Advances in RNA research could pave new way for development of anti-cancer drugs

Research findings point to potential new targeted therapy for SMA

Scientists find new target that may lead to future treatments for Kennedy's disease

HMS study reveals key instigator of nerve cell damage in ALS patients

First investigational treatment for infantile-onset SMA shows promising results in clinical trial

Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

Using NMR to investigate intrinsically disordered proteins: an interview with Dr Isabella Felli

New paediatric exoskeleton aims to support children with spinal muscular atrophy

Bereaved parents feel that pediatric end-of-life care needs improvement

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Gene-replacement therapy could be potential treatment option for SMARD1

Long-term exercise may benefit Spinal Muscular Atrophy patients

Dr. Elliot J. Androphy receives IGNITE grant to develop drug candidate for spinal muscular atrophy

Neuroscientists identify specific enzyme that plays critical role in spinal muscular atrophy

Synthetic RNA and DNA could reverse protein deficiency that causes Friedreich's ataxia

'Housekeeping' gene may have a link to male infertility

Alternative splicing: a new approach to drug development? An interview with Lucy Donaldson

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Advancing Intracellular Delivery with LumiPore®

How ELRIG Is Helping Mid-Career Scientists Navigate Leadership, Career Change and Redundancy

The role of UV-Vis in biopharma QC

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