080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Archive for December, 2010

ADDEX PHA : récompensé par le prix EuropaBio de la PME de Biotechnologie la plus innovante en Europe

Zonebourse.com |


Les modulateurs allostériques représentent une approche radicalement nouvelle et inexploitée en chimie, par rapport aux médicaments traditionnels, et peuvent rivaliser avec des médicaments à base de protéines. Ces petites molécules à visée thérapeutique, actives par voie orale pourraient offrir aux patients de meilleurs résultats que les médicaments classiques, tous les deux étant des thérapies de petites molécules et à base de protéines. Et, plus important, elles peuvent à la fois agir plus précisément sur leur récepteur cible de l’organisme, et en même temps, permettre un contrôle plus précis de la fonction du récepteur. En effet, elles se fixent aux récepteurs de surface cellulaire sur un site différent de celui sur lequel se fixent les médicaments traditionnels. Bien que les sites de fixation allostériques offrent un avantage certain, il a été difficile jusqu’à présent d’identifier les molécules qui se fixent sur ces sites en utilisant les techniques conventionnelles de criblage à haut débit. Addex a réuni une bibliothèque unique de composés allostériques ainsi que des tests biologiques propriétaires de criblage à haut débit, qui rendent possible la recherche de modulateurs allostériques à une échelle industrielle.


Fragile X Therapeutics Programs Reviewed by NeuroInvestment

CARDIFF, CA–(Marketwire – December 1, 2010) – NI Research has released the December issue of NeuroInvestment, which features a comprehensive review of therapeutics for the treatment of Fragile X.

Fragile X has become a focus for a growing number of pharmaceutical companies as they have considered the potential value of addressing an orphan disorder which is a significant neurodevelopmental disorder in its own right, the single most common form of inherited mental disability/retardation. Successful disease-modification in Fragile X may also be the gateway to disease-modifying treatments for autism, and perhaps even schizophrenia.


The Fragile X mutation leads to the underproduction of FMRP, an essential mediator of synaptic protein synthesis, leading to defects in both structure and cognitive function. The major approaches to Fragile X, beyond the crude attempts at neuroleptic symptom control currently available, include mGluR5 antagonism (Seaside Therapeutics, Novartis, Roche), GABA-B antagonism (Seaside Therapeutics), and PAK inhibition (Afraxis). Other potential strategies include neurotrophic/protective drugs (Neuren, Cortex Pharmaceuticals) and oxytocin (Cypress Bioscience). Hint of concept has been established in pilot studies conducted by Novartis and Seaside Therapeutics.


TIME.com | New Version of an Old Drug Could Treat Autism (and Addiction Too)

Après le New York Times, le magazine Forbes, au tour du TIME magazine : tour du chapeau pour les avancées scientifiques ET cliniques dans le domaine du X fragile ! ! !

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Maia Szalavitz, Time.com |

One night in 2006, Kathy Roberts rushed her autistic daughter, Jenny, to the hospital. Nothing had been able to stop the young woman, then in her mid-20s, from vomiting. Jenny had recently suffered several major seizures and her entire gastrointestinal system was going haywire.

To try to calm Jenny’s GI tract, doctors at Massachusetts General Hospital prescribed baclofen, an antispasmodic drug that is also being studied as a potential treatment for alcoholism and other addictions. The drug relieved Jenny’s vomiting, but it did something else too — a completely unexpected and welcome side effect. (More on TIME.com: Could Anorexia Be a ‘Female’ Form of Autism?)

“Within 24 hours, I saw a change,” says Roberts. “Right away, I saw that it was globally calming. I’ve always described a state that she would get into where it seemed like she wasn’t comfortable in her own skin, and was trying to crawl out. I saw that calmed down.”

Roberts, founder of the Giant Step school for children with autism in Southport, Conn., called Mark Bear, professor of neuroscience at MIT and advisory board member of Giant Step. In 2005, Bear had co-founded a drug company called Seaside Therapeutics to develop treatments for autism and other developmental disorders. Roberts told Bear about baclofen’s effect on her daughter, and a new line of research was born. (More on TIME.com: Picky Eating May Be Early Sign of Autism)