You Searched For "national policy for treatment of rare diseases" and got 15 results
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2 mins read
Aetna will now pay for more kids with a devastating rare disease to get a $2.1 million drug, reversing earlier denials
"While the data on Zolgensma is limited, emerging information, expert opinions and the promise of t…
Emma Court
2 mins read
My baby has a rare disease. Hearing from others that I am a great mom keeps me going.
The author shared her feelings while her baby was in the hospital and said it put into perspective …
J.B. Lacombe
2 mins read
The FDA just approved a new treatment for a devastating disease. The price tag is more than $2 million.
The treatment, Novartis's Zolgensma, is a one-time therapy that works to treat the disease at the g…
Emma Court
8 mins read
Million-dollar drugs are on the way to treat rare and devastating diseases. The US is scrambling to figure out how to pay for them.
Pharma giants are pouring billions into the promising space. In the last week alone, Swiss drug gia…
Emma Court
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
2 mins read
Here's how to treat Stiff Person Syndrome, the neurological condition that Céline Dion was diagnosed with that affects her voice
Céline Dion announced on Thursday that she had been diagnosed with Stiff Person Syndrome, a neurolo…
Yeji Jesse Lee
2 mins read
$300,000 A Year 'Orphan Drugs' Are Becoming A Hugely Profitable Business
Much is riding on the drug’s success: Gattex is the centerpiece of a comeback plan for NPS, which n…
Max Nisen
2 mins read
A company that calls itself the 'Flatiron Health of rare diseases' and is backed by Mark Zuckerberg, Bill Gates, and Jeff Bezos just raised $3 million
Faber then teamed up with former 23andMe executive Nancy Yu to found RDMD, a platform that works wi…
Charlotte Hu
2 mins read
We got an exclusive look at the pitch deck used by AI health company Healx to raise a $56 million Series B
"Originally we weren't planning on doing a Series B this year but Atomico approached us and we were…
Callum Burroughs,Callum Burro…
3 mins read
Girl with rare deadly disease receives revolutionary million-dollar gene-therapy drug – but it is too late for her older sister
Young sisters Teddi and Nala Shaw from the UK both have late-infantile MLD, an inherited genetic di…
Isobel van Hagen
9 mins read
'Like we were being forced to gamble with our son's life': Health insurers won't pay for a $2.1 million drug for kids, and parents say they're running out of time
Gene therapies are cutting-edge treatments that could make big differences for debilitating disease…
Emma Court
1 min read
Ovid Therapeutics shares fall 28% as investors question new drug data
The drug showed a favorable safety profile and was tolerated in adults and adolescents with the dis…
Charlotte Hu
5 mins read
A top executive at Swiss drug giant Novartis told us the inside story of the $2.1 million price tag for the most expensive drug in the world
Payers have also been very interested in agreements where a portion of the price tag is returned in…
Emma Court
2 mins read
Kids with 'bubble boy' disease could finally get access to a life-saving gene therapy, as a study starts back up at UCLA
While families who have kids with the disease are desperate for the therapy, drug companies have be…
Andrew Dunn
3 mins read
What to know about Kawasaki disease, a rare condition in children that doctors worry is linked to the coronavirus
100 children in Europe, and at least 25 children in New York City, have been hospitalized with symp…