Morning Rounds: Preterm birth rates vary across the U.S., with the highest in the Southeast
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Category: rare diseases
STAT+: Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy
The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell…
CRISPR is about to make history. Here’s the 5-minute rundown of what to expect
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New gene therapies confront many sickle cell patients with an impossible choice: a cure or fertility
As a teenager, Marie Tornyenu was always having to explain herself. If it wasn’t the…
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STAT+: Official: FDA needs staff influx to meet gene therapy needs
WASHINGTON — The Food and Drug Administration needs dozens more reviewers if it wants its…
STAT Morning Rounds: Six health news stories you need to start your day
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STAT+: How sickle cell became the first disease treated by CRISPR
Nearly a decade ago, consultants delivered to Rodger Novak a kind of Sears catalog of…
STAT+: After four patients died of gene therapy, Astellas sees possible explanation — and a way forward
A gene therapy study, halted when four children with a rare neuromuscular disorder died after…
The first vaccine for the increasingly prevalent chikungunya virus is here
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Opinion: State Prescription Drug Affordability Boards could end up limiting access to the life-saving cystic fibrosis medication Trikafta
I was living on the precipice of end-stage disease when I enrolled in the clinical…
Opinion: How the government can help lower the price — not just the cost — of cutting-edge gene therapies
At the bustling Chicago O’Hare airport, my luggage and I collided with an elderly couple….
Your Monday morning biotech news roundup
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STAT+: ALS patients support bill that could help patients with other rare diseases
WASHINGTON — A month after advisers to the Food and Drug Administration overwhelmingly voted against…
WHO considers adding ‘noma,’ a rare childhood disease, to its list of neglected conditions
It’s a disease of children living in extreme poverty. Its cause is uncertain. So is…
Why Biogen is purposefully becoming more risk averse, according to Biogen’s CEO
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STAT+: Peter Marks on creating Operation Warp Speed, but for rare diseases
Peter Marks wants drug developers to ask more stupid questions. It’s part of the top…
With Ozempic, Wall Street moves on from a whole other class of medicines
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With a rare immunodeficiency disease, a performer and patient advocate learned lessons the hard way
When asked where he’s from, Casey Warford often waffles. He was born in San Antonio,…
STAT+: Mirum’s Chenodal succeeds in late-stage trial for rare metabolic disorder
Mirum Pharmaceuticals reported on Monday that a drug it recently acquired to treat cerebrotendinous xanthomatosis,…
STAT+: Taysha Gene Therapies’ plan to tackle rare diseases is crumbling
In 2020, as biotech stocks surged amid the pandemic, a startup called Taysha Gene Therapies…
Opinion: STAT+: The IRA needs changes to better support patients with rare diseases
This time last year, members of Congress were working around the clock to draft and…
STAT+: Neurocrine Biosciences drug for congenital adrenal hyperplasia succeeds in late-stage trial
SAN DIEGO — Neurocrine Biosciences announced on Tuesday that an experimental drug for congenital adrenal…
STAT+: Crinetics acromegaly drug succeeds in late-stage trial but would face stiff competition if approved
SAN DIEGO — Crinetics Pharmaceuticals announced on Sunday that its experimental drug for acromegaly, a…
An uncommon thyroid diagnosis led to an unexpected, all-too-visible side effect
Annie Larsson, 40, is no stranger to chronic conditions. She was diagnosed with type 1…
STAT+: AstraZeneca sues to immediately stop Medicare drug price negotiation program
WASHINGTON — AstraZeneca is the sixth drug maker to pile on a legal challenge to…
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Travere Therapeutics drug, approved for one rare and deadly kidney disease, fails in study of another
SAN DIEGO — Travere Therapeutics, a biotech focused on treating rare diseases, on Monday announced…
STAT+: Pharmalittle: Advisory panel narrowly votes down ALS drug; Vertex looks to advance experimental pain drug
Good morning. Elizabeth Cooney here, borrowing the virtual Pharmalittle chair while Ed Silverman takes Manhattan…
STAT+: New CRISPR tools could fix diseases caused by large DNA rearrangements, scientists report
Ever since the now Nobel-winning discovery of CRISPR genome editing in 2020, scientists have been…
STAT+: After repeated losses in the U.S., Catalyst wins a victory in Canada over a rare disease drug
In an unexpected move, Canada’s Federal Court quashed the approval of a rare disease drug…
STAT Plus: Pharmalittle: White House signs deal for U.S.-made generics to fight Covid-19; Gilead expands remdesivir donations
Rise and shine, another brand new day is here. Thanks to a sunny sky and…
STAT Plus: ‘It’s the disparity in who gets affected’: A sickle cell doctor tracks dollars and drug development
Sickle cell disease and cystic fibrosis are both rare, genetic diseases. While there are three…
When the hoofbeats really are a zebra’s, a patient community helps me navigate a new rare-disease reality
“I have some unexpected news.” That’s what the doctor said to me when my 10-month-old…
STAT Plus: College student with disease that causes premature aging is heartened by medical advances
In many ways, Emerson College freshman Meghan Waldron seems like a lot of students in…