Sentences with phrase «dystrophy drug»

«New muscular dystrophy drug target identified.»

PTCT, +3.33 % plans to buy privately - held Marathon Pharmaceuticals» controversial Duchenne muscular dystrophy drug for about $ 75 million in cash and $ 65 million in PTC common stock.

Sarepta continued to rise after it received approval from the FDA earlier this week for a muscular dystrophy drug.

Sarepta Therapeutics, which won a pioneering Food and Drug Administration approval for its Duchenne muscular dystrophy drug last year, has settled a patent dispute with rival BioMarin over the «exon - skipping» technology at heart of the companies» muscular dystrophy treatments.

Not exact matches

The saga of Sarepta Therapeutics» rare disease drug Exondys 51, the first - ever treatment for the degenerative movement disorder Duchenne muscular dystrophy, has taken yet another turn.

When Sarepta Therapeutics nabbed a pioneering approval for a Duchenne muscular dystrophy (DMD) drug last year, it won more than just bragging rights and a controversial FDA approval: the firm also landed a coveted «priority review voucher,» which can either be sold off to another company or used to slash the review period for a future Sarepta treatment.

Shares of PTC Therapeutics, Inc. (NASDAQ: PTCT) gained more than 13 percent Friday after the FDA found that the company's experimental drug to treat Duchenne muscular dystrophy may work.

She has been part of various charities and fundraising events, including Kids Escaping Drugs, Say Yes, Parent - Child Connection, Hamburg Finance Academy, New York State Association of Counties, and the Muscular Dystrophy Association.

This time, parents of children with muscular dystrophy are battling the US medicines regulator, the Food and Drug Administration (FDA).

Drugs that turn on PGC - 1beta could be used to treat muscle - wasting diseases like muscular dystrophy — or to create superathletes.

A study published by scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham provides insight into the mechanism of action of the drug ataluren, which is showing promise in treating Duchenne muscular dystrophy and cystic fibrosis.

Following up on these promising results, they decided to explore whether the drug SR8278 could slow the progression of muscular dystrophy in an animal model.

Judy Anderson, a muscular dystrophy expert at the University of Manitoba in Winnipeg, Canada, adds that evidence for acetylcholine defects in human muscular dystrophy has been contradictory over the years, but if this preliminary finding holds up in mice and humans, it could pave the way for new drug targets.

«Drugs with multiple targets show promise against myotonic dystrophy type 1.»

«The identification of these genes paves the way for the development of a test to identify individuals who are at risk of getting Fuchs dystrophy, and also for the development of drugs to slow disease progression or to potentially delay or prevent it from occurring,» said George A. McKie, D.V.M., PhD., director of the NEI's cornea research program.

Laboratory fruit flies are used for quick screening of candidate drug treatments for Duchenne muscular dystrophy.

Drugs targeting myostatin could prove a godsend for people with muscle - wasting diseases such as muscular dystrophy.

Now a scientist reports that mice engineered to make extra follistatin, which deactivates myostatin, have four times the muscle of regular mice, suggesting a new target for drugs to fight muscle - wasting diseases such as muscular dystrophy.

Oligonucleotide treatments recently have been approved by the Food and Drug Administration for two neuromuscular diseases: Duchenne's muscular dystrophy and spinal muscular atrophy (SMA).

Drugs tested by the U-M appear to correct the signaling pathway that is disrupted in muscular dystrophy at an earlier step than the phosphodiesterase inhibitors.

A preclinical study led by researchers in the United States has found that a new oral drug shows early promise for the treatment of muscular dystrophy.

In addition to supercharging stamina, the drug, called AICAR, may also be useful in treating debilitating muscular disorders such as muscular dystrophy as well as metabolic diseases such as diabetes, because it also appears to help the body use and remove sugar from the blood more effectively.

«Cancer drug shows promise in reducing toxic genetic material in myotonic dystrophy.»

Scientists also tested the drug in a mouse model of muscular dystrophy, since prednisone is normally given for this disease.

They found the same drug will also switch off similar signals in a protein implicated in Duchenne Muscular Dystrophy (DMD).

We are going to screen a larger library of chemicals to identify molecules that either boost or weaken NMD, which should help develop better and more targeted drugs for treating ALS, muscular dystrophy and cystic fibrosis.»

Validating this original concept, we previously demonstrated that PGD - derived hES cells and their derivatives, which express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), offer pertinent disease - cell models, applicable for a wide systemic mechanistic analysis ranging from functional studies at the cellular level to a large - scale drug screening.

Current development - stage programs include antisense drugs to treat SMA, ISIS - SMNRx, and myotonic dystrophy type 1, ISIS - DMPKRx.

«We see real promise in therapeutic strategies for SMA that increase production of the SMN protein,» said Muscular Dystrophy Association Executive Vice President Research and Medical Director Valerie Cwik, M.D. «We're delighted ISIS Pharmaceuticals is moving forward with a Phase 1 dose - escalation study of its antisense drug in children with SMA.»

Speakers said that drug development for muscle - wasting SMA offers promise not only for those with this disease, but also possibly for patients with other diseases such as amyotrophic lateral sclerosis (ALS), muscular dystrophy, and Parkinson disease.

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UPDATE 9/9/16: The SRPT stock jumped in price to over $ 53 a share today (9/19/16) on the news that Sarepta's drug, Exondys 51, which treats Duchenne muscular dystrophy has been given accelerated approval.

The company was listed as a big buy in many spots because of a positive meeting they just had with the FDA in regards to the approval of their new drug to treat Muscular Dystrophy.

Breed - Related Health Concerns Allergies to certain drugs such as insecticides and flea treatments; seizures; axonal dystrophy (disease of the nerves and muscles)