You Searched For "duchenne muscular dystrophy" and got 20 results
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1 min read
Delhi HC exempts custom duties on medicines for rare diseases
The Delhi High Court has said that custom duties and charges will not be applied to medicines, drug…
2 mins read
Sarepta tumbles after the FDA rejects its muscular dystrophy treatment - a decision that left the CEO 'very surprised'
The company's first drug for Duchenne muscular dystrophy was approved in 2016 after prolonged debat…
Ben Winck
3 mins read
Govt waives clinical trial requirement for several drugs approved in select countries
This waiver would ensure expeditious availability of the latest medicines for treating diseases …
PTI
1 min read
Centre gives full basic customs duty exemption on import of drugs for rare diseases
In a significant step, the Centre has given full exemption from basic customs duty on all drugs as …
3 mins read
5L patients of rare disease, treatment costs in crores; govt lacks budget: HC told
The court had earlier asked the ministry to write to the company -- Sarepta Therapeutics in USA -- …
PTI
1 min read
Sarepta Therapeutics spikes 57% after a key trial shows its experimental gene therapy worked in 3 patients
"I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels…
Jonathan Garber,Lydia Ramsey
9 mins read
Some patients say a commonly prescribed steroid triggered mania and suicidal ideation. I should know — it happened to me.
Prednisone is prescribed to millions of Americans. While mood swings are a known side effect, some …
Julia Métraux
2 mins read
An old steroid used to treat a rare disease just got approved by the FDA - and it costs $89,000
The move is reminiscent of a plan former pharma CEO Martin Shkreli had with a treatment for another…
Lydia Ramsey,Reuters
2 mins read
Another controversial drugmaker just left the industry's most powerful lobby
PhRMA has made a point of distinguishing itself from companies that have drawn outrage about drug p…
Lydia Ramsey
2 mins read
Biotech stock explodes 80% after the FDA approves the first drug to treat a rare muscle disorder
"Accelerated approval makes this drug available to patients based on initial data, but we eagerly a…
Akin Oyedele
2 mins read
Biotech stock crashes 50% after disclosing bad news from the FDA
"The advisory committee voted 3 - 7, with three abstentions, against finding substantial evidence b…
Akin Oyedele
4 mins read
Over 1,500 federal workers affected by the government shutdown are asking for donations on GoFundMe to help pay their bills
Nikolai, Niko for short, has Muscular Dystrophy Duchenne, a disease that left him wheelchair-bound,…
Mariana Alfaro
2 mins read
STOCKS GO NOWHERE: Here's what you need to know
An early rally in West Texas Intermediate crude futures also fizzled out. Venezuelan President Nico…
Akin Oyedele
2 mins read
This stainless steel wristwatch just became the most expensive ever sold
The model is a variation on Patek Phillipe's 5016 model, except that it's made with a stainless ste…
Dennis Green
3 mins read
Congress is going after another drugmaker for pricing a decades-old drug at $89,000
"We believe Marathon is abusing our nation's "orphan drug" program, which grants companies seven ye…
Lydia Ramsey
1 min read
Biotech Stock Crashes After FDA Requires Additional Data For New Drug Application
In a statement Chris Garabedian, president and CEO of Sarepta Therapeutics said: "We are committed …
Myles Udland
2 mins read
Paramount Goes Above And Beyond To Help A Sick 'Teenage Mutant Ninja Turtles' Fan
What a spectacular trend this has become. Do you recall, recently, when Marvel went out of its way …
Sean O'Connell
3 mins read
10 things you need to know before European markets open
Brits are holding more and more cash, and it is a worrying sign for the UK's post-Brexit economy, a…
Will Martin
3 mins read
LeBron James sent a heartfelt letter to a 19-year-old with a terminal illness
I've heard some pretty amazing things about you, and from what I've heard, I can tell you are an un…
Scott Davis
3 mins read
Sarepta Is Surging After Good News For Its Ebola Treatment
AVI-6002 for the treatment of Ebola is a combination therapy of two phosphorodiamidate morpholino o…