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Orphan Disease Center establishes new initiative that focuses on ALS

Orphan Disease Center establishes new initiative that focuses on ALS

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established a new Program of Excellence for Motor Neuron Disease. [More]
Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy. [More]
CSHL researcher-inventor hails FDA approval of new SMA drug

CSHL researcher-inventor hails FDA approval of new SMA drug

Within a week of Christmas day, a drug called nusinersen will be in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). [More]
Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. [More]
MDA celebrates FDA approval of new spinal muscular atrophy drug

MDA celebrates FDA approval of new spinal muscular atrophy drug

The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants. [More]
FDA approves first drug to treat children and adults with spinal muscular atrophy

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. [More]
Advances in RNA research could pave new way for development of anti-cancer drugs

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. [More]
Research findings point to potential new targeted therapy for SMA

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. [More]
Scientists find new target that may lead to future treatments for Kennedy's disease

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. [More]
HMS study reveals key instigator of nerve cell damage in ALS patients

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. [More]
First investigational treatment for infantile-onset SMA shows promising results in clinical trial

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. [More]
Promising new therapeutic target could lead to better prognosis of spinal muscular atrophy

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. [More]
Using NMR to investigate intrinsically disordered proteins: an interview with Dr Isabella Felli

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. [More]
New paediatric exoskeleton aims to support children with spinal muscular atrophy

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. [More]
Bereaved parents feel that pediatric end-of-life care needs improvement

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. [More]
Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

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. [More]
Gene-replacement therapy could be potential treatment option for SMARD1

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. [More]
Long-term exercise may benefit Spinal Muscular Atrophy patients

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

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. [More]
Neuroscientists identify specific enzyme that plays critical role in spinal muscular atrophy

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

Neuroscientists have discovered a specific enzyme that plays a critical role in spinal muscular atrophy, and that suppressing this enzyme's activity, could markedly reduce the disease's severity and improve patients' lifestyles. [More]
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