Navigation Links
Study shows promise for new drug to treat Fragile X
Date:1/7/2011

The first drug to treat the underlying disorder instead of the symptoms of Fragile X, the most common cause of inherited intellectual disability, shows some promise according to a new study published in the January 5 issue of Science Translational Medicine. Researchers from Rush University Medical Center helped design the study and are now participating in the larger follow-up clinical trial.

The data from the early trial of 30 Fragile X patients, found the drug, called AFQ056, made by Novartis Pharmaceuticals, helped improve symptoms in some patients. Patients who had the best response have a kind of "fingerprint" in their DNA that could act as a marker to determine who should get treatment.

"This is an exciting development. It is the first time we have a treatment targeted to the underlying disorder, as opposed to supportive treatment of the behavioral symptoms, in a developmental brain disorder causing intellectual disability. This drug could be a model for treatment of other disorders such as autism," said pediatric neurologist Dr. Elizabeth Berry-Kravis, a study author and director of the Fragile X Clinic and Research Program and the Fragile X-Associated Disorders Program at Rush.

The drug is designed to block the activity of mGluR5, a receptor protein on brain cells that is involved in most aspects of normal brain function, including regulation of the strength of brain connections, a key process required for learning and memory. Fragile X patients have a mutation in a single gene, known as Fragile X Mental Retardation-1 or FMR1. The mutation prevents FMR1 from making its protein, called FMRP, such that FMRP is missing in the brain. FMRP normally acts as a blocker or "brake" for brain cell pathways activated by mGluR5. When FMRP is missing, mGluR5 pathways are overactive resulting in abnormal connections in the brain and the behavioral and cognitive impairments associated with Fragile X.

The research team, led b
'/>"/>

Contact: Kim Waterman
Kimberly_Waterman@rush.edu
312-942-7820
Rush University Medical Center
Source:Eurekalert

Page: 1 2

Related medicine news :

1. Age Takes Toll on Facial Bones, Study Finds
2. Circumcision Helps Cut HPV Transmission Rate, Study Finds
3. Liver disease a possible predictor of stroke: Study
4. Neural stem cells maintain high levels of reactive oxygen species, UCLA study finds
5. Study of sarcoid-like granulomatous pulmonary disease finds elevated rates in WTC responders
6. Doctor Behind Study Linking Vaccine to Autism Accused of Deliberate Fraud
7. Study Links Obesity to Greater Pain, Weakness in Fibromyalgia Patients
8. Loss of gene promotes brain-tumor development, reduces survival, study finds
9. Infant Organ Donors Could Help Meet Transplant Needs: Study
10. Study Sheds Light on Genetic Risk of MS
11. Vaccine Protects Mice From Cocaines Effects, Study Finds
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/6/2015)... Wheaton, Illinois (PRWEB) May 06, 2015 RPS ... new take on the tried and true Rock Paper Scissors ... , With fun characters that encourage storytelling and easy to ... the new twist on an old favorite. RPS Revolution ... transforms it into whimsical characters that are grouped in set ...
(Date:5/6/2015)... 06, 2015 As Hilo Medical ... EMR Adoption Model, the hospital looked for new ... and automate processes. The leadership team realized that ... electronic workflows, patient registration still required patients to ... With HIMSS Stage 7 attestation fast approaching, Hilo ...
(Date:5/6/2015)... (PRWEB) May 06, 2015 Corporate ... behalf of MEBC, an Alliance Award for excellence ... during FOCUS 2015, JDA’s annual user conference which ... retail and wholesale distribution sectors. This award recognizes ... are receiving services that advance their supply chain ...
(Date:5/6/2015)... The Beryl Institute announces the publication of ... (PXJ), an international, open access, peer-reviewed journal focused on ... experience. The second volume of PXJ supports the goal ... the voices engaged and provides the space for listening, ... The Beryl Institute, Volume 2, Issue 1 includes ...
(Date:5/6/2015)... (PRWEB) May 06, 2015 Marketing ... #1 ranked international water, sanitation and hygiene nonprofit by ... communities across the world and it brings our agency ... CEO of Marketing Maven. “The ongoing relationship between ... available for people to learn about and support the ...
Breaking Medicine News(10 mins):Health News:RPS Revolution Launches Kickstarter Campaign 2Health News:RPS Revolution Launches Kickstarter Campaign 3Health News:Access Electronic Patient Signatures Help Hilo Meet HIMSS Stage 7 Paperless Targets & Save $200,000 a Year on Consent Forms 2Health News:Access Electronic Patient Signatures Help Hilo Meet HIMSS Stage 7 Paperless Targets & Save $200,000 a Year on Consent Forms 3Health News:MEBC Receives Award for Consulting Excellence in Retail 2Health News:Patient Experience Journal Releases Spring 2015 Issue 2Health News:Marketing Maven Expands Partnership with the #1 Ranked International Water, Sanitation and Hygiene Nonprofit 2Health News:Marketing Maven Expands Partnership with the #1 Ranked International Water, Sanitation and Hygiene Nonprofit 3
... ... the Obama White House is counting on support from union members as it makes its ... International/O,Leary Report Poll, rank and file union members are not sold on many of the ... ...
... , , , , ... announces the grand opening of its 116th dental practice, Coast Dental St. Lucie ... West Boulevard. The Lucie County Chamber of Commerce will conduct a ... by Panera Bread((R)). The public is invited to meet Dr. Quinton ...
... , , SAN DIEGO, Sept. 22 ... device company, held its Annual Meeting of the Shareholders for the fiscal ... Del Mar Hilton in Del Mar, California. The business matters considered and ... number of directors of the Company at six and election of six ...
... Program at Georgetown University Medical Center are now recruiting volunteers ... of its kind for the treatment of patients with dementia ... study examines the safety and possible benefits of CERE-110. ... a part of the brain affected by Alzheimer,s disease. ...
... Academy Development Initiative (ASADI) will be held Nov. 9-11 in ... Ghana Academy of Arts and Sciences. The theme of ... health in Africa, which will be discussed by top experts ... with the release of Science in Action: Saving the Lives ...
... , , SALT LAKE ... ) announced it has signed a preferred vendor agreement to ... Network, an independent physical therapy, occupational therapy and speech-language pathology ... Colorado. , , "We,re pleased ...
Cached Medicine News:Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 2Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 3Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 4Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 5Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 6Health News:Union Members Reject Key Tenets of "Obamacare" - According to Recent Zogby/O'Leary Poll 7Health News:Coast Dental Opens New Office in St. Lucie West September 28 2Health News:SpectraScience Announces Results of Annual Shareholder Meeting 2Health News:SpectraScience Announces Results of Annual Shareholder Meeting 3Health News:Historic gene therapy trial to treat Alzheimer's disease underway at Georgetown 2Health News:Dynatronics to Supply Western Rehabilitation Health Network's 114 Clinics 2
(Date:5/6/2015)... -- Alexza Pharmaceuticals, Inc. (Nasdaq: ALXA ) today ... 2015.  The net loss and the loss from operations ... million, respectively, compared to $10.7 million and $8.8 million ... 31, 2015, Alexza had consolidated cash, cash equivalents, marketable ... "We continue to focus on ways in which to ...
(Date:5/6/2015)... 6, 2015 /CNW/ - Celebrities, bloggers and influencers across ... spread the important message that melanoma prevention begins with parents-encouraging ... Sharapova , Hilary Duff , and parenting author ... to their networks on Monday to share this poignant video ... The David Cornfield Melanoma Fund (DCMF)-producers of ...
(Date:5/6/2015)... 2015 Research and Markets ( ... "Cannulas Market by Product (Nasal, Arterial, Arthroscopy, ... Material (Plastic, Metal) & End Users (Hospitals, Ambulatory ... report to their offering. The global ... CAGR of 5.6% from 2015 to 2020 to ...
Breaking Medicine Technology:Alexza Reports 2015 First Quarter Financial Results 2Alexza Reports 2015 First Quarter Financial Results 3Alexza Reports 2015 First Quarter Financial Results 4Alexza Reports 2015 First Quarter Financial Results 5Alexza Reports 2015 First Quarter Financial Results 6Alexza Reports 2015 First Quarter Financial Results 7Alexza Reports 2015 First Quarter Financial Results 8Alexza Reports 2015 First Quarter Financial Results 9Alexza Reports 2015 First Quarter Financial Results 10Celebrities Tweet Their Support of Melanoma Awareness Month and #newfamilyrule 2Global Cannulas Market 2015 - Forecasts to 2020 with Medtronic, Edward Lifesciences, Terumo, Sorin & Maquet Holding B.V. & Co. Dominating 2Global Cannulas Market 2015 - Forecasts to 2020 with Medtronic, Edward Lifesciences, Terumo, Sorin & Maquet Holding B.V. & Co. Dominating 3
... 7 In response to intense speculation and supposition ... (Pink Sheets: ASFX) has scheduled a conference call for ... management will provide a general corporate update. , The ... access code is 453492250#. It will be recorded ...
... DNA testing for gene variants that can increase the ... the anti-clotting drug Plavix® (clopidogrel bisulfate) is now being ... Scripps Health patients electing to undergo coronary stent procedures. ... system in the U.S. to deploy this genetic testing ...
Cached Medicine Technology:Quest Diagnostics Brings Genetic Testing for Plavix(R) Response to Coronary Stent Patients at Scripps Health 2Quest Diagnostics Brings Genetic Testing for Plavix(R) Response to Coronary Stent Patients at Scripps Health 3
... matrix constructed of cross-linked collagen fibers ... through a proprietary 360 accretion process. ... aspirate, Healos provides an excellent environment ... differentiation. The hydroxyapatite-coated collagen microfibers in ...
Sharp Weitlaner Retractor....
Weitlaner retractor, blunt....
Bruns (Volkmann) bone curette, with Novotex plastic handle (autoclavable in autoclave up to 143C by 3 bar), 9, oval....
Medicine Products: