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As it was reported in SCIENCE, they could snip out a faulty segment (exon) of the gene in mice. They have not yet replace the gap with a normal exon. It is more difficult.
[[ ... The red-hot genome editing tool known as CRISPR has scored another achievement: Researchers have used it to treat a severe form of muscular dystrophy in mice. Three groups report today in Science that they wielded CRISPR to snip out part of a defective gene in mice with Duchenne muscular dystrophy (DMD), allowing the animals to make an essential muscle protein. The approach is the first time CRISPR has been successfully delivered throughout the body to treat grown animals with a genetic disease...]]
Hope for Muscular Dystrophy patients After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerful new gene-editing technique.
Sure sure buddy.
sell it or go buy another stock. We dont need your comments ok. Go buy some twtr
A paragraph from an article commenting on the award: [[... Their work grew out of a finding that bacteria could remember viruses. Researchers had found segments of viral genes, sandwiched between repeated bacterial DNA sequences. These viral scraps serve as an infection memory bank: From them, bacteria create guide RNAs that can seek out the DNA of returning viruses before chopping up the viral genes with a nuclease, Cas9. Once this mechanism was understood, Doudna and Charpentier, among others, raced to adapt it to editing DNA in higher organisms.]]
These people who applied the principle to higher organisms would certainly gain the Nobel prize in near future.
When RNAi was chosen for Nobel prize in physiology in 2006, $ALNY took off from low teens to 20s. Vertex may rise too.
Yahoo will not allow posting cut and paste from the tumbler web site. You can find the blog under "dreaming of breathing" when you type orkambi blog in the google search bar.
Actually side effect and drop out rate (about 4%) are about the same as in TRAFFIC and TRANSPORT Phase 3 trials on which FDA approval was based for Orkambi. Patients are aware that adverse reactions usually go away over a period of weeks and patients are significantly improved thereafter in rates of serious infections weight gain, quality of life, and re-hospitalization. As a results, the great majority of CF patients and their caregivers are prepared for tolerating the initial adverse respiratory effects noted in the first weeks of Orkambi treatment as the patients mobilize the chronic infected secretions in their lungs, and finally benefit from the drugs effects in reversing the underlying cause of the illness by improving CFTR function. The blog below is from a young adult CF patient that exemplifies the Orkambi experience in the majority of CF patients.
Chest tightness, short of breath, constant nausea, coughing up blood, headaches. The drug should have NEVER been approved and there was conflicting information from the beginning. It was marginally approved because of crying parents who wanted something, but now patients are dropping off, afraid to start or continue even after a month.
Nice to get this recommendation going into the JP Morgan healthcare investor conf in three weeks
[[ JPMorgan’s Cory Kasimov and team name Biogen (BIIB), Vertex Pharmaceuticals (VRTX), and BioMarin Pharmaceutical (BMRN) their top biotech picks for 2016.]]
[[ ... we see Vertex as a very attractive investment with commercial momentum and potential bottom line leverage combined with a substantial amount of meaningful data and significant strategic value.]]
"Roll up and gouge for same medication" Wall Street loves your plan.
May all of your new years be audited and audited on every level possible. Just think how many people needing medical care got billed all year long, you deserve auditing at the company and personal levels.
Pennyvulture, you seem ill informed about a lot of things. To start with Harvoni costs about 100k for a 12 week course of treatment, HCV is known for destroying the liver, not kidneys, and for most, there are no ill effects until the majority of the liver has been badly damaged by scar tissue. I see your stance on social issues, justice and politics have a similarly ill informed sound to them, but this in not the board to get into that.
Anytime a lib like you mentions the word "justice", all I hear is lazy people who want the govt to give you things by taking them away from others.
Think about this...in the police killing in Chicago, the city gave the parents $5M for their loss. In almost any fatal loss, the value of a human life is placed at $1M by actuaries.
So GILD spends $15B, develops a drug that CURES (ie, doesn't treat, it cures) HCV, sells it for under $40K, and they get plastered in the press and by the politicians.
People who have used that drug were living miserable, useless lives, often on dialysis (costly), and headed to the kidney waiting list, where they would probably end up dying bcse that list is so long.
But $37K is too high priced? To get your life back?
Good suggestion. I'll own many common shares when option expiration dates come in future months.
Lentiglobin is working well if beta-thalassemia patients do NOT have beta 0 homozygous genotype. Even for the homozygotes benefit is there. For SCD, more data are need to determine the value of the procedure. It appears that for some patients it is working; it may be age-related, or partial bone marrow cell ablation is the cause of poor results for others.