Tag Archives: neurodegenerative disease

April, 2018

25 April
Ionis’ Investigational Huntington’s Disease Drug Demonstrates Lowering of the Disease-Causing Protein and Improvement in Clinical Measures in Phase 1/2 Study

CARLSBAD, Calif., April 24, 2018 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today presented top-line data from the Phase 1/2 study of IONIS-HTTRx (RG6042) in people with early stage Huntington’s disease (HD) at the 70th American Academy of Neurology (AAN) meeting in Los Angeles, California. …
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10 April
Novartis to Acquire Gene Therapy Company AveXis for $8.7 Billion

Basel, April 9, 2018 – Novartis announced today that it has entered into an agreement and plan of merger with AveXis, Inc. to acquire the US-based Nasdaq-listed clinical stage gene therapy company for USD 218 per share or a total of USD 8.7 billion in cash. The transaction was unanimously approved by …
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14 July
New Imaging Technique Able to Watch Molecular Dynamics of Neurodegenerative Diseases

WASHINGTON — Researchers have developed a fast and practical molecular-scale imaging technique that could let scientists view never-before-seen dynamics of biological processes involved in neurodegenerative diseases such as Alzheimer’s disease and multiple sclerosis. The new technique reveals a sample’s chemical makeup as well as the orientation of molecules making up …
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8 May
FDA Approves MT Pharma America’s Radicava for Treatment of Amyotrophic Lateral Sclerosis

JERSEY CITY, N.J., May 5, 2017 – MT Pharma America, Inc. today announced the U.S. Food and Drug Administration (FDA) has granted approval of RADICAVA™ (edaravone), as an intravenous infusion treatment for amyotrophic lateral sclerosis (ALS), a rapidly progressive neurodegenerative disease in which the majority of patients die within two …
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21 October
FDA Accepts Resubmission of Teva’s Huntington Disease Drug

JERUSALEM–(BUSINESS WIRE)–Teva Pharmaceutical Industries Ltd. (NYSE and TASE:TEVA) today announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the New Drug Application (NDA) for SD-809 (deutetrabenazine) for the treatment of chorea associated with Huntington disease (HD). The FDA has assigned a Prescription Drug User Fee …
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15 August
New Protein Target Emerges for C9orf72 the Most Common Genetic Form of ALS

WASHINGTON, Aug. 12, 2016 /PRNewswire-USNewswire/ — Targeting a single protein, called SUPT4H1, reduces the levels of the three toxic entities created by the C9orf72 gene expansion, the most common genetic cause of ALS, according to a study published in the journal Science. The finding suggests that SUPT4H1 may be a …
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14 October
Takeda and UCL Enter Research Partnership to Identify Novel Target Genes for Neurodegenerative Disease

LONDON & CAMBRIDGE, England–(BUSINESS WIRE)–UCL (University College London) and Takeda Pharmaceutical Company Limited (TSE:4502) today announced a new research collaboration to identify and validate novel target genes for the treatment of neurodegenerative disease. This collaboration will focus on mechanistic approaches for the identification of genes or signalling pathways that modify …
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