psalexa
logo

Fragile-x Syndrome Therapeutics Pipeline Analysis

Fragile-x Syndrome Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments

Delivery : 1-2 Working Days
Report Code: LS10953
Available Format:

Due to lack of awareness among people regarding genetic disorders which leads to mutations in one’s DNA, the prevalence of fragile-x syndrome is increasing, across the globe. Smoking, alcohol consumption during pregnancy and hereditary disorders are few of the primary reasons to for the increase in prevalence of fragile-x syndrome. The therapeutic pipeline of fragile-x syndrome is expected to grow more rapidly in the future on account of active involvement of pharmaceuticals & biotechnology companies carrying out research programs to develop effective therapeutic agents or drugs for eradicating fragile-x syndrome. According to a study conducted by Centers for Disease Control and Prevention (CDC) in 2012, around one female in every 151 females & one male in every 468 males in the U.S., was affected by fragile-x syndrome. Moreover, according to the study, occurrence of fragile-x syndrome is higher in females as compared to males.  

Fragile-x syndrome is a genetic disorder that affects the development and learning capabilities of an individual. It is also called as Marker-x syndrome and Martin-Bell syndrome. Fragile-x syndrome causes trinucleotide repeat in Fragile-x mental retardation 1(FMR-1) gene present on X chromosome. This gene is responsible for making a protein called Fragile-x mental retardation protein (FMRP), which is required for normal neural development. In case of an individual suffering from fragile-x syndrome, production of FMRP is constrained due to the repetition of CGG (trinucleotide) in FMR1 gene. The symptoms associated with fragile-x syndrome are mental retardation, learning disability, impulsiveness, stuttering, development delays, seizures, depression, hyperactivity, etc.

The therapeutic pipeline of fragile-x syndrome includes some of the important therapeutic agents or drugs are available under the pipeline of different pharmaceutical companies. OV-101 is a drug being currently developing by Ovid therapeutics Inc., and is under Phase I stage of clinical trials. The drug, OV-101, is orally administered, and targets GABA-A (gamma-aminobutyric acid) receptor delta (GABRD) & agonist to GABA-A receptor delta (GABRD). Bellus Health Inc. in collaboration with AMO Pharmaceutical, is developing a drug by the name, AMO-01, for the treatment of fragile-x syndrome. It is currently under Phase II stage of clinical trials. AMO-01 is a small molecule which targets peripheral benzodiazepine receptor (PBR). Ganaxolone, a drug being currently developed by Marinus pharmaceutical, is under Phase II clinical trials and is orally administered. Ganaxolone is a small molecule which modulates gamma-aminobutyric acid (GABA). NNZ-2566 is a drug being developed by Neuren Pharmaceuticals, which shows the efficacy to treat Rett syndrome and Fragile-x syndrome. NNZ-2566 is under Phase II clinical trials and inhibits the activity of cytokines in the human body.

Some of the companies having a pipeline of Fragile-x syndrome therapeutics include Ovid therapeutics, Marinus pharmaceuticals, Neuren pharmaceuticals, Bellus health Inc., AMO pharmaceuticals, Eli-lly& co., Anavex Life Sciences Corp., Kareus Therapeutics, and Zynerba Pharmaceuticals, Inc.

Place An Order

Single User License
question-mark

The report will be delivered in PDF format without printing rights. It is recommended for a single user.

USD 3250
Group License
question-mark

The report will be delivered in PDF format along with the printing rights. It is recommended for up to five users.

USD 4000
Enterprise License
question-mark

The report will be delivered in PDF format along with printing rights and detailed Excel sheet. It is recommended for organizations where multiple people would like to access the report from multiple locations.

USD 7750

Pre-Purchase Enquiry