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Home » Keywords » spinocerebellar ataxia

Items Tagged with 'spinocerebellar ataxia'

ARTICLES

Cerebellum, brain stem, spinal cord
Neurology/psychiatric

Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3

May 15, 2025
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The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).
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Tree in the shape of human head losing leaves
Neurology/psychiatric

Systemic CAG-targeted shRNA approach reduces mutant protein and aggregates in humanized polyQ mice

March 24, 2025
Polyglutamine (polyQ) diseases, a group of dominantly inherited CNS disorders, are caused by an abnormal expansion of cytosine-adenine-guanine repeats (usually over 35-40 repeats). PolyQ diseases, including spinocerebellar ataxia and Huntington’s disease, cause brain neurodegeneration, leading to progressive motor and often cognitive signs.
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Illustration of double helix
Neurology/psychiatric

Sarepta Therapeutics licenses programs from Arrowhead Pharmaceuticals

Nov. 27, 2024
Sarepta Therapeutics Inc. has signed a licensing and collaboration agreement with Arrowhead Pharmaceuticals Inc. to obtain exclusive global rights to multiple clinical, preclinical and discovery-stage programs for rare genetic diseases of the muscle, central nervous system (CNS) and the lungs. The deal includes ARO-DUX4, ARO-DM1, ARO-MMP7 and ARO-ATXN2.
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Sarepta ‘secures future’ with massive Arrowhead collaboration

Nov. 26, 2024
By Jennifer Boggs
In the largest collaboration of 2024, Sarepta Therapeutics Inc. and Arrowhead Pharmaceuticals Inc. entered a sprawling global licensing deal that includes a swath of clinical and preclinical candidates targeting rare genetic diseases. Under the terms, Sarepta gains access to existing and potential future compounds derived from the RNAi platform developed by Arrowhead, with the latter eligible for payments potentially exceeding $11 billion.
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Biohaven's phase III rare disease data bode well for troriluzole

Sep. 23, 2024
By Lee Landenberger
A little more than a year after the U.S. FDA refused to review the NDA for Biohaven Ltd.’s ultra rare disease treatment, new and positive phase III data have changed the treatment’s momentum. The upbeat results came as a surprise to analysts and investors, with the stock having a strong day and the company prepping an NDA for a fourth quarter submission.
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FDA refuses to review NDA for Biohaven’s ultra-rare disease treatment

July 27, 2023
By Lee Landenberger
Not hitting the primary endpoint of a phase III study caused the U.S. FDA to say it would not review Biohaven Ltd.’s NDA for troriluzole to treat spinocerebellar ataxia (SCA), an ultra-rare disorder.
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Genetic brain disorders, conceptual 3D illustration
Neurology/Psychiatric

Spinocerebellar ataxia variants show vast heterogeneity

June 14, 2023
By Mar de Miguel
The phenotypic variety of spinocerebellar ataxias (SCAs) not caused by CAG repeat expansion (polyglutamine SCA) is greater than expected. A collaboration directed by scientists of the Paris Brain Institute described seven variants of this disorder in 756 individuals, observing that age at onset and progression by gene and variant can occur from childhood to late adulthood with very different forms of the disease.
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Cerebellum, brain stem, spinal cord

Reprocell reports mixed results for phase II Stemchymal trial in spinocerebellar ataxia

May 30, 2023
By Tamra Sami
Regenerative medicine product Stemchymal, an allogeneic adipose-derived mesenchymal stem cell treatment, missed the primary efficacy endpoints in two phase II spinocerebellar ataxia trials conducted in Japan and Korea, but a subpopulation analysis showed efficacy signals in patients with more severe conditions, Reprocell Inc. reported.
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Kavli Prize in Neuroscience medal

Kavli Neuroscience Prize honors four for genetic discoveries

June 2, 2022
By Anette Breindl
Four scientists have shared the 2022 Kavli Prize in neuroscience, "for pioneering the discovery of genes underlying a range of serious brain disorders," together and separately.
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Handshake dollar sign

Cadent acquired by Novartis in a $770M deal

Dec. 17, 2020
By Lee Landenberger
By acquiring privately held Cadent Therapeutics Inc., Novartis AG gains full rights to Cadent’s allosteric modulator program, part of which the two companies began collaborating on in 2015, and all the company’s outstanding stock. Cambridge, Mass.-based Cadent will receive as much as $770 million, $210 million of it up front, plus $560 million in milestones.
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