Muscular Atrophy News and Research

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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.

25 Sep 2015

Long-term disease progression in spinocerebellar ataxia quantified

Researchers have quantified the progression of the most common spinocerebellar ataxias over a follow-up period of up to 8 years in steps to improve sample size calculations for future interventional trials.

16 Sep 2015

Special issue of Future Science OA highlights research on protein misfolding diseases

Future Science Group today announced the publication of a special issue in Future Science OA, covering the rapidly evolving field of protein misfolding diseases.

28 Aug 2015

Determining the dynein-dynactin complex structure: an interview with Dr Gabriel C. Lander

This is a macromolecular assembly is made up of two components, dynein and dynactin, that works to move molecular cargo (organelles, RNA, vesicles, proteins, viruses) along microtubule highways within our cells.

16 Aug 2015

Researchers report role of two gene-regulating molecules in Parkinson's disease

As Parkinson's disease progresses in patients, a puzzling dichotomy plays out in their brains. One set of neurons degenerates, while a similar population nearby is spared the same degree of damage. Why the difference? An answer to this question could clear the way for preventions and treatments for this disease, which impairs movement.

11 Aug 2015

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

AstraZeneca and Isis Pharmaceuticals, Inc. today announced a strategic collaboration to discover and develop antisense therapies for cardiovascular, metabolic and renal diseases.

3 Aug 2015

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

Isis Pharmaceuticals, Inc., the leader in RNA-targeted therapeutics, and Akcea Therapeutics, its wholly owned subsidiary, announced today that The New England Journal of Medicine (NEJM) has published positive clinical results from a Phase 2 clinical study evaluating volanesorsen (formerly ISIS-APOCIII Rx) in patients with very high to severely high triglycerides.

30 Jul 2015

Researchers use gene-editing technique involving low-dose irradiation to repair human stem cells

For the first time, researchers have employed a gene-editing technique involving low-dose irradiation to repair patient cells, according to a study published in the journal Stem Cells Translational Medicine. This method, developed by researchers in the Cedars-Sinai Board of Governors Regenerative Medicine Institute, is 10 times more effective than techniques currently in use.

23 Jul 2015

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals, Inc. announced today that it has earned a $2.15 million milestone payment from Biogen related to advancing the ongoing pivotal Phase 3 study (CHERISH) evaluating ISIS-SMN Rx in children with spinal muscular atrophy (SMA).

14 Jul 2015

Akcea Therapeutics obtains FDA Orphan Drug Designation for volanesorsen

Akcea Therapeutics, a wholly-owned subsidiary of Isis Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS).

6 Jul 2015

Researchers use pioneering stem cell techniques to better understand causes of Spinal Muscular Atrophy

New research from the Advanced Gene and Cell Therapy Lab at Royal Holloway, University of London has used pioneering stem cell techniques to better understand why certain cells are more at risk of degenerating in Spinal Muscular Atrophy than others.

1 Jul 2015

Study on fruit flies, brewer's yeast may provide clues about cause of Spinal Muscular Atrophy

Scientists at the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) have shown that fruit flies and brewer's yeast can reveal clues about the cause of Spinal Muscular Atrophy (SMA), the most common genetic killer of infants.

30 Jun 2015

Innovative gene transfer-based treatment for kids with giant axonal neuropathy

University of North Carolina School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN).

26 Jun 2015

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on children with spinal muscular atrophy (SMA) who have completed the open-label, Phase 2 multiple-dose study of ISIS-SMN Rx and are continuing to receive treatment in an open-label extension (OLE) study.

22 Jun 2015

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMN Rx in infants with Type I spinal muscular atrophy (SMA).

11 Jun 2015

Cytokinetics, The ALS Association expand partnership to fight against ALS

Cytokinetics, Incorporated and The ALS Association announced an expanded partnership in which the company will provide Gold Level Sponsorship of the National Walks to Defeat ALS as well as Platinum Level Sponsorship for ALS Association Golden West Chapter initiatives.

4 Jun 2015

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Good Start Genetics, Inc., a commercial-stage molecular genetics information company, today presented data from two studies analyzing detection rates among patients at in vitro fertilization (IVF) clinics who underwent carrier screening for inherited diseases. The studies were presented at the 2015 Annual Clinical and Scientific Meeting of The American College of Obstetricians and Gynecologists being held in San Francisco, Calif.

7 May 2015

Proteomics helps identify previously unrecognized proteins and pathways in nerve regeneration

Using proteomics techniques to study injured optic nerves, researchers at Boston Children's Hospital have identified previously unrecognized proteins and pathways involved in nerve regeneration. Adding back one of these proteins--the oncogene c-myc--they achieved unprecedented optic nerve regeneration in mice when combined with two other known strategies.

5 May 2015

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-ANGPTL3Rx. In this study, healthy volunteers treated with ISIS-ANGPTL3Rx achieved dose-dependent, statistically significant reductions in angiopoietin-like 3 (ANGPTL3) of up to 93 percent with a mean reduction of up to 84 percent from baseline (p<0.001).

23 Mar 2015

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE).

24 Feb 2015