Saturday, August 23, 2014

Study confirms close link between immune system and adult neurogenesis

Study confirms close link between immune system and adult neurogenesis. News Medical, Published on August 19, 2014.

these findings would stimulate a new area of research into immune therapies to target neurological diseases

More patients help drug companies pass valley of death - Indianapolis Business Journal

More patients help drug companies pass valley of death - Indianapolis Business Journal

But patient groups—especially those formed around rare and untreatable diseases—are indeed playing a crucial role in getting experimental drugs across the proverbial “valley of death”—where none of those deep-pocketed players want to put their money.

Intellect Neurosciences, Inc. Issues Letter to Shareholders - GlobeNewswire press release

Intellect Neurosciences, Inc. Issues Letter to Shareholders - GlobeNewswire press release

Shire is developing OX1 as a treatment for Friedreich's Ataxia and possibly other diseases for which OX1 may qualify for orphan drug designation.

Grant Alert: Hematopoietic Stem Cell-Based Therapy For Friedrich Ataxia (University Of California San Diego, La Jolla, United States)

Grant Alert: Hematopoietic Stem Cell-Based Therapy For Friedrich Ataxia (University Of California San Diego, La Jolla, United States). Project Leader: CHERQUI, STEPHANIE

The role of iron in neurodegenerative disorders: insights and opportunities with synchrotron light

The role of iron in neurodegenerative disorders: insights and opportunities with synchrotron light. Collingwood Joanna Frances, Davidson Mark Rogers, Front. Pharmacol., 19 August 2014 | doi: 10.3389/fphar.2014.00191

FULL TEXT PDF

Friday, August 22, 2014

Preimplantation genetic diagnosis for inherited neurological disorders

Preimplantation genetic diagnosis for inherited neurological disorders. Ilan Tur-Kaspa, Roohi Jeelani
& P. Murali Doraiswamy, Nature Reviews Neurology 10, 417–424 (2014), doi:10.1038/nrneurol.2014.84

Preimplantation genetic diagnosis (PGD) is an option for couples at risk of having offspring with an inherited debilitating or fatal neurological disorder who wish to conceive a healthy child.

The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children

The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children. Annemarie Rosan Kreeftmeijer-Vegter, Anthonius de Boer, Roselinda H van der Vlugt-Meijer and Peter J de Vries; Orphanet Journal of Rare Diseases 2014, 9:120 doi:10.1186/s13023-014-0120-x

OPEN ACCESS; FULL TEXT PDF

Wednesday, August 13, 2014

STATegics has identified CNS-available small molecule erythropoietin (EPO) Allomimetics that selectively activate the tissue-protective EPO receptor

STATegics has identified CNS-available small molecule erythropoietin (EPO) Allomimetics that selectively activate the
tissue-protective EPO receptor
.

The compounds also demonstrate a favorable safety profile and penetrance to the brain at pharmacologically relevant concentrations, and are being investigated for treatment of PD, Friedreich’s ataxia, and acute kidney injury.

Tuesday, August 12, 2014

Electrochemical impedance sensor based on small molecule modified Au electrode for the recognition of a trinucleotide repeats

Electrochemical impedance sensor based on small molecule modified Au electrode for the recognition of a trinucleotide repeats .
Hanping He, Xiaoqian Peng, Min Huang, Gang Chang, Xiuhua Zhang and Shengfu Wang; Analyst, 2014, Accepted Manuscript,
DOI: 10.1039/C4AN00853G; Received 12 May 2014, Accepted 10 Aug 2014

The electrochemical impedance sensor provides the simple and rapid method to detect trinucleotide repeats without requiring labelling and immobilizations of DNA, making it promising for the early diagnosis of the neurodegenerative diseases

Deferiprone in Friedreich’s Ataxia: A six-month randomized controlled trial

Deferiprone in Friedreich’s Ataxia: A six-month randomized controlled trial. Massimo Pandolfo, Javier Arpa, Martin B. Delatycki, Kim Hanh Le Quan Sang, Caterina Mariotti, Arnold Munnich, Irene Sanz-Gallego, Geneieve Tai, Mark A. Tarnopolsky, Franco Taroni, Michael Spino and Fernando Tricta; Annals of Neurology, DOI: 10.1002/ana.24248. Accepted manuscript online: 11 AUG 2014 09:41AM EST | DOI: 10.1002/ana.24248