Spinal Muscular Atrophy News and Research

RSS

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.

Stem cells from patients offer model, drug-discovery platform for early-onset form of Alzheimer's

Harvard stem cell scientists have successfully converted skins cells from patients with early-onset Alzheimer's into the types of neurons that are affected by the disease, making it possible for the first time to study this leading form of dementia in living human cells. This may also make it possible to develop therapies far more quickly and accurately than before.

5 Mar 2014

Isis Pharmaceuticals provides update on ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA).

25 Feb 2014

FDA approves CSC’s Phase II clinical trial for cancer immunotherapy in women with ovarian cancer

California Stem Cell, Inc. (CSC) announced today that the U.S. Food and Drug Administration (FDA) has approved the Company's application to begin a Phase II clinical trial exploring the potential of a patient-specific cancer immunotherapy in women with Stage III or IV ovarian, fallopian tube or primary peritoneal cancer.

19 Feb 2014

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin.

11 Feb 2014

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received new awards from the California Institute of Regenerative Medicine (CIRM), the state stem cell research agency, that will forward revolutionary stem cell science in medicine.

30 Jan 2014

Joint research program in SMA enters initial stage of clinical development

PTC Therapeutics, Inc., the SMA Foundation, and Roche, announced today that their joint research program in Spinal Muscular Atrophy has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers.

22 Jan 2014

NUS scientists open possible new route for treatment of spinal muscular atrophy

A recent study led by scientists from the National University of Singapore (NUS) opens a possible new route for treatment of Spinal Muscular Atrophy (SMA), a devastating disease that is the most common genetic cause of infant death and also affects young adults.

18 Dec 2013

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

The exact definition of orphan diseases varies depending on where you are in the world. In the EU it's defined as a prevalence of less than 1 in 2,000, which equates to about 250,000 patients across the EU.

4 Dec 2013

Principal investigators at Nationwide Children's Hospital elected as AAAS Fellows

Brian K. Kaspar, PhD, and Veronica J. Vieland, PhD, principal investigators in The Research Institute at Nationwide Children's Hospital, have been named Fellows of the American Association for the Advancement of Science (AAAS).

26 Nov 2013

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy.

5 Nov 2013

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children's Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA).

19 Oct 2013

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and the Muscular Dystrophy Association (MDA) today announced a new partnership to develop and implement the U.S. Neuromuscular Disease Registry, a patient registry that will play an important role in determining effective treatments for people with muscular dystrophy and related muscle diseases.

9 Oct 2013

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA).

4 Oct 2013

Final patient completion of phase II study of TR040303 in patients treated for acute myocardial infarction announced by Trophos

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today the final patient completion of a phase II study of TR040303 in patients treated for acute myocardial infarction (MI). Outcome data of this study is expected to be available towards the end of 2013.

2 Oct 2013

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA.

25 Sep 2013

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.

19 Sep 2013

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

A research team at Cold Spring Harbor Laboratory has used a recently developed technology they call TSUNAMI to create the first animal model of the adult-onset version of spinal muscular atrophy, a devastating motor-neuron illness.

10 Sep 2013

One-time treatment slows onset and progression of ALS, study finds

Studies of a therapy designed to treat amyotrophic lateral sclerosis suggest that the treatment dramatically slows onset and progression of the deadly disease, one of the most common neuromuscular disorders in the world.

10 Sep 2013

Biogen Idec And Isis Ink Fourth Deal In Two Years

...

9 Sep 2013

Neurodyn acquires all assets related to new drug candidate Memogain from Galantos Pharma

Neurodyn Inc. announced today that it has acquired from Galantos Pharma GmbH, Mainz, Germany all assets related to their Alzheimer's prescription drug candidate - Memogain. Memogain has completed an extensive preclinical development program and is ready to proceed into Phase 1 clinical trials in late 2013. The terms of the acquisition were not disclosed.

5 Sep 2013