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

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

Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich's ataxia, a neurological disease for which there is currently no cure. [More]
'Housekeeping' gene may have a link to male infertility

'Housekeeping' gene may have a link to male infertility

Researchers at Iowa State University have found evidence that a "housekeeping" gene present in every cell of the body may have a link to male infertility. [More]
Alternative splicing: a new approach to drug development? An interview with Lucy Donaldson

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

RNA is becoming an interesting drug target as it takes possible intervention back one step to the synthesis of a target protein, instead of trying to block or inhibit a process. [More]
FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

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

Ionis Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to IONIS-HTTRx for the treatment of patients with Huntington's disease (HD). [More]
Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

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

Isis Pharmaceuticals, Inc. today announced that its partner, Biogen, has initiated a Phase 1/2 clinical study of ISIS-SOD1 Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS). [More]
UF Health investigators make new discovery about Huntington's disease

UF Health investigators make new discovery about Huntington's disease

University of Florida Health researchers have made a new discovery about Huntington's disease, showing that the gene that causes the fatal disorder makes an unexpected "cocktail" of mutant proteins that accumulate in the brain. [More]
MedUni Vienna researchers discover genetic cause of a rare disease

MedUni Vienna researchers discover genetic cause of a rare disease

Researchers at MedUni Vienna have discovered the genetic cause of a rare disease characterized by life-threatening "liver crises" in early childhood and subsequent manifestation of neurological symptoms, such as neuropathy and ataxia (a movement disorder), when they reach school age. [More]
Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study evaluating ISIS-FXIRx in patients with end-stage renal disease (ESRD) on hemodialysis. In May 2015, Isis entered into an exclusive license agreement with Bayer HealthCare (Bayer) to develop and commercialize ISIS-FXIRx for the prevention of clotting disorders. [More]
Genetic carrier screening: an interview with Don Hardison, CEO of Good Start Genetics

Genetic carrier screening: an interview with Don Hardison, CEO of Good Start Genetics

Carrier screening provides you and your doctor with information about your risks of having a child with a serious genetic disease prior to or during pregnancy. [More]
Clinical data from spinal muscular atrophy program presented at 20th International WMS Congress

Clinical data from spinal muscular atrophy program presented at 20th International WMS Congress

PTC Therapeutics, Inc., today announced that clinical data from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) were presented at the 20th International Congress of the World Muscle Society (WMS) in Brighton, U.K. [More]
HSCI researchers find surprising similarities between SMA and ALS

HSCI researchers find surprising similarities between SMA and ALS

Harvard Stem Cell Institute researchers studying spinal muscular atrophy (SMA) have found what they term "surprising similarities" between this childhood disorder that attacks motor neurons and amyotrophic lateral sclerosis (ALS), more commonly known as Lou Gehrig's disease. [More]
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