SOUTH PLAINFIELD, N.J., Aug. 23, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that it has successfully completed the acquisition of Agilis Biotherapeutics, Inc., a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous system (CNS).
The acquisition also includes gene therapy programs in development, GT-FA, GT-AS, and GT-RLN, for Friedreich Ataxia, Angelman Syndrome and Cognitive Disorders associated with several neurodevelopmental and neurodegenerative disorders, respectively.
Thursday, August 23, 2018
Longitudinal Diffusion Tensor Imaging in the brain in Friedreich’s Ataxia: follow-up at 12 and 24 months
Pierre-Gilles Henry, James Joers, Dinesh Deelchand, Diane Hutter, and Christophe Lenglet; International Society for Magnetic Resonance in Medicine, (Abstract #3730) 16-21 June 2018
We report 12-month and 24-month longitudinal diffusion tensor imaging (DTI) data in the brain of subjects with Friedreich’s ataxia (FRDA). Significant longitudinal changes were observed in several brain areas (including the corpus callosum, internal capsule and superior corona radiata) in a group of 13 patients over 24 months. Our data suggest that diffusion MRI of the brain could be useful to better understand the impact of FRDA on brain microstructure and connectivity, and to assess the effect of potential treatments on neurodegeneration in upcoming clinical trials in FRDA.
We report 12-month and 24-month longitudinal diffusion tensor imaging (DTI) data in the brain of subjects with Friedreich’s ataxia (FRDA). Significant longitudinal changes were observed in several brain areas (including the corpus callosum, internal capsule and superior corona radiata) in a group of 13 patients over 24 months. Our data suggest that diffusion MRI of the brain could be useful to better understand the impact of FRDA on brain microstructure and connectivity, and to assess the effect of potential treatments on neurodegeneration in upcoming clinical trials in FRDA.
Noninvasive brain stimulation may help treat symptoms of rare movement disorders
August 22, 2018, American Academy of Neurology.
Electrical stimulation of the brain and spinal cord may help treat the symptoms of rare movement disorders called neurodegenerative ataxias, according to a study published in the August 22, 2018, online issue of Neurology, the medical journal of the American Academy of Neurology. There are several types of these disorders, which can be hereditary or occur randomly, including spinocerebellar ataxia, multiple system atrophy and Friedreich's ataxia.
Electrical stimulation of the brain and spinal cord may help treat the symptoms of rare movement disorders called neurodegenerative ataxias, according to a study published in the August 22, 2018, online issue of Neurology, the medical journal of the American Academy of Neurology. There are several types of these disorders, which can be hereditary or occur randomly, including spinocerebellar ataxia, multiple system atrophy and Friedreich's ataxia.
Public summary of opinion on orphan designation: Omaveloxolone for treatment of Friedreich's ataxia
EMA/395368/2018, 16 August 2018
On 27 June 2018, orphan designation (EU/3/18/2037) was granted by the European Commission to Dr Stefan Blesse, Germany, for omaveloxolone (also known as RTA 408) for the treatment of Friedreich’s ataxia.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 24 May 2018 recommending the granting of this designation.
On 27 June 2018, orphan designation (EU/3/18/2037) was granted by the European Commission to Dr Stefan Blesse, Germany, for omaveloxolone (also known as RTA 408) for the treatment of Friedreich’s ataxia.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 24 May 2018 recommending the granting of this designation.
Jupiter Orphan Therapeutics Announces Favorable Data Which Expands JOTROL's Applications to the Estimated $5 Billion Mitochondrial Rare Disease Market
JUPITER, Fla., Aug. 22, 2018 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc. ("JOT"), Jupiter, FL, today announced that it has received new data regarding an increase in mitochondrial biogenesis in liver and brain from a recently performed study in a frequently studied Alzheimer's disease animal model, the triple transgenic (APPsw / PS1M146V / TauP301L) mouse.
Murdoch Children's Research Institute, a JOT partner, has conducted an open label study showing that high dose of resveratrol (not JOTROL), with associated GI-side effects, had significant positive impact in 4 vital endpoints. Target, for this indication, is to reproduce this study with JOTROL and reproduce the same positive results without side effects.
Murdoch Children's Research Institute, a JOT partner, has conducted an open label study showing that high dose of resveratrol (not JOTROL), with associated GI-side effects, had significant positive impact in 4 vital endpoints. Target, for this indication, is to reproduce this study with JOTROL and reproduce the same positive results without side effects.
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