I mean the government does......short and long term taxes for 2014 already started early..
then if you can do it 6 more times like this, wow at the taxes..
always some excuse to run a sell market and the same for the bull market,
if it is played well this year the gov could make 2 trillion in taxes for 2014 in the market alone....or more
stem cells in ''10-15 years good
a regeneration of a bad gene is a cure but could cause the fall of other genes that are weak that work with one or another....blocks of repaired or replaced genes would be the ticket.. or multi-gene replacement and that is down the road 10-15 years..
Remember this,,,, Changing the Natural history of the DMD disease is the key of approval and sarepta did this......anyway the fda feels good about compiling evidence to make them happy is fine..''no other company but sarepta has done this.
government tax income...............as soon as the market sells down about 5- 7 percent it will be a drive back up......
Jan 24 (Reuters) - PTC Therapeutics Inc said a committee of the European Medicines Agency did not recommend conditional approval of its drug for the treatment of a rare muscular disorder, sending its shares down as much as 28 percent in premarket trading.
The company said it intends to request the committee for a re-examination. It was seeking a so-called conditional approval, granted to treatments that show early benefit. The drugmaker still needs to conduct trials.
The drug, ataluren, is intended to treat a form of Duchenne muscular dystrophy which occurs due to a type of genetic mutation known as a nonsense mutation.
Proprietary, Advanced Generation PMO-Based Chemistries
Sarepta has dedicated significant resources to the development of advanced generation RNA-based antisense chemistries with superior drug-like performance characteristics. This investment has been productive and we are currently applying several novel and proprietary PMO based platform chemistries to the discovery and development of new drug candidates. Specifically, we have advanced our foundational PMO chemistry through the construction of a series of novel and proprietary PMO-based platforms.
Sarepta’s most recent advance in chemistry, PMO-X, is focused on the incorporation of new proprietary chemical technology to fine-tune important physicochemical properties, intended to enhance in vivo tissue targeting, selectivity, and potency. The intrinsic neutral structure of the PMO provides continuing opportunities to create innovative and potentially transformative RNA-based therapeutics with varied beneficial properties.
Another proprietary analogue platform, our PMOplus™, uses the addition of positionally specific molecular charges into the PMO backbone. This is intended to specifically enhance drug performance characteristics on two key parameters: targeted cell penetration, and the maintenance of antiviral performance in the presence of viral mutation.
Sarepta has developed a proprietary technology of peptide conjugated phosphorodiamidate morpholino oligomers, or our PPMO platform. Using our PPMO technology, cellular uptake of the active PMOs, as well as their potency and specificity of tissue targeting, are significantly enhanced by the conjugation of arginine-rich cell-penetrating peptide chemical moieties (CPPs) to a PMO.
details more on this one..
Hutchinson-Gilford progeria syndrome is a genetic condition characterized by the dramatic, rapid appearance of aging beginning in childhood. Affected children typically look normal at birth and in early infancy, but then grow more slowly than other children and do not gain weight at the expected rate (failure to thrive). They develop a characteristic facial appearance including prominent eyes, a thin nose with a beaked tip, thin lips, a small chin, and protruding ears. Hutchinson-Gilford progeria syndrome also causes hair loss (alopecia), aged-looking skin, joint abnormalities, and a loss of fat under the skin (subcutaneous fat). This condition does not disrupt intellectual development or the development of motor skills such as sitting, standing, and walking.
People with Hutchinson-Gilford progeria syndrome experience severe hardening of the arteries (arteriosclerosis) beginning in childhood. This condition greatly increases the chances having a heart attack or stroke at a young age. These serious complications can worsen over time and are life-threatening for affected individuals.
How common is Hutchinson-Gilford progeria syndrome?
This condition is very rare; it is reported to occur in 1 in 4 million newborns worldwide. More than 130 cases have been reported in the scientific literature since the condition was first described in 1886.
What genes are related to Hutchinson-Gilford progeria syndrome?
Mutations in the LMNA gene cause Hutchinson-Gilford progeria syndrome.
The LMNA gene provides instructions for making a protein called lamin A. This protein plays an important role in determining the shape of the nucleus within cells. It is an essential scaffolding (supporting) component of the nuclear envelope, which is the membrane that surrounds the nucleus. Mutations that cause Hutchinson-Gilford progeria syndrome result in the production of an abnormal version of the lamin A
The why for detail--
Mutations in LMNA have been described for a wide variety of diseases including Emery-Dreifuss muscular dystrophy, dilated cardiomyopathy with or without conduction system disease, limb-girdle muscular dystrophy type 1B, Dunnigan-type familial partial lipodystrophy, mandibuloacral dysplasia, Charcot-Marie-Tooth neuropathy type 2B1, Hutchinson-Gilford progeria syndrome and atypical Werner syndrome. These mutations include missense, nonsense, splicing, and deletion mutations. The pathogenesis of these mutations is still being investigated.
The John Welsh Cardiovascular Diagnostic Laboratory offers molecular genetic testing for LMNA mutations. Individuals are tested by automatic fluorescent DNA sequencing of all 12 exons of the LMNA gene. We strongly recommend initial testing of an affected individual, if available, in order to provide the greatest test sensitivity and clearest interpretation of results for subsequent family members. Genetic counseling is recommended for all individuals in order to identify additional at-risk family members and to discuss reproductive issues.
Reasons for Referral
Molecular confirmation of the diagnosis of Emery-Dreifuss muscular dystrophy, dilated cardiomyopathy with or without conduction system disease, limb-girdle muscular dystrophy type 1B, Dunnigan-type familial partial lipodystrophy, mandibuloacral dysplasia, Charcot-Marie-Tooth neuropathy type 2B1, Hutchinson-Gilford progeria syndrome, or atypical Werner syndrome
Genomic DNA is analyzed for LMNA mutations by automatic fluorescent DNA sequencing of all 12 exons of the LMNA gene, as well as the exon/intron junctions and a portion of the 5′ and 3′ untranslated regions. Patient DNA is sequenced in both the forward and reverse orientations. If a mutation is identified, additional family members are analyzed only for the familial mutation(s) by automatic fluorescent DNA
What is Muscular Dystrophy?
The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.
Duchenne MD is the most common form of MD and primarily affects boys. It is caused by the absence of dystrophin, a protein involved in maintaining the integrity of muscle. Onset is between 3 and 5 years and the disorder progresses rapidly. Most boys are unable to walk by age 12, and later need a respirator to breathe. Girls in these families have a 50 percent chance of inheriting and passing the defective gene to their children. Boys with Becker MD (very similar to but less severe than Duchenne MD) have faulty or not enough dystrophin.
Facioscapulohumeral MD usually begins in the teenage years. It causes progressive weakness in muscles of the face, arms, legs, and around the shoulders and chest. It progresses slowly and can vary in symptoms from mild to disabling.
Myotonic MD is the disorder's most common adult form and is typified by prolonged muscle spasms, cataracts, cardiac abnormalities, and endocrine disturbances. Individuals with myotonic MD have long, thin faces, drooping eyelids, and a swan-like neck.
people are being added to healthcare like crazy over the next 10 years and the aging population...
common sense ..I see biotech growth like the software growth was.New medicines will replace old medicines .