Politico24d
An opening for an ultra-rare disease drug
That’s a longstanding issue with rare-disease drug candidates, given the inherently small patient base available for trials. McCarthy said the panel’s input would be helpful as the FDA ...
The Boston Globe5d
FDA approves Boston biotech’s drug for ultra-rare disease of immune system
The Food and Drug Administration has approved a Boston biotech’s drug for an ultra-rare disease of the immune system that leaves patients more susceptible to potentially life-threatening ...
pharmaphorum1y
ultra-rare diseases
The UK's regulator has granted early access to Alnylam's ultra-rare disease drug lumasiran, allowing patients with primary hyperoxaluria type 1 to gain access ahead of a regulatory decision ...
talker on MSN1mon
Ultra-rare upside-down birth of bat captured on camera for the first time
The birth of an ultra-rare bat which was born upside down has been captured on camera for the first time. Footage shows the ...
The Sun26d
‘Statement of luxury’, inside British car brand’s ‘ultra-rare’ new model with just 10 being made…& quaint town behind it
RANGE Rover has released an 'ultra rare' new model that's inspired by a quaint Cotswold town. There are just 10 Range Rover SV Burford Editions in existence, with the exclusive 4x4 described as a ...
Yahoo1mon
This Ultra-Rare Porsche 911 Was the First of Its Kind in the U.S. and Now It’s up for Grabs
A rare 1982 911 Turbo Slantnose is currently up ... but customization divisions are nothing new. Porsche’s ultra-exclusive Sonderwunsch special requests program has been catering to the marque ...
CNN24d
Ultra-rare, bizarre-looking blind mole photographed in Australia
Editor’s Note: Call to Earth is a CNN editorial series committed to reporting on the environmental challenges facing our planet, together with the solutions. Rolex’s Perpetual Planet ...
Yahoo10d
This Ultra-Rare McLaren F1 Could Fetch Over $20 Million at Auction
You haven’t been able to buy a brand-new McLaren F1 since the late 1990s, but an upcoming auction offers the next best thing. Next month, Sotheby’s Sealed will sell the only example of the ...
UPI5d
Researchers identify genetic cause of rare movement disorder
Researchers have conclusively identified the genetic cause of a rare, progressive movement disorder. A rare extra-long version of a gene appears to cause nerve cells to become poisoned by toxic ...
New York Post4d
AstraZeneca cops to rare, deadly side effect of COVID jab as lawsuits mount
AstraZeneca has admitted that its COVID-19 vaccine could cause a rare but deadly blood-clotting condition, potentially exposing the UK pharmaceutical giant to tens of millions in lawsuits brought ...
Forbes2mon
February 29 Has Been A Rare Yet Interesting Date In Baseball Finance
So it is “rare ... 29, 2016, in Cleveland where ownership opened the purse strings for a different $4 million payout. It all went to 37-year-old infielder Juan Uribe. He hit .206 in 73 ...