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5-29-2019 zolgensma

$2.1 million gene therapy is most expensive drug in history

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the ...
frase

How this promising gene therapy for a rare neuromuscular disease was fueled by passionate parents and a dog

The gene therapy will be too late for Joshua Frase. But it will be his legacy ...
4-29-2019 dims

Why we shouldn’t view gene therapy as a treatment of last resort

Providers and payers must ask themselves a two-pronged question about gene therapy: Who should be treated and when? … The ...
4-18-2019 unnamed file

Gene therapy’s fight against ‘bubble boy’ disease may have yielded a safe cure

“Cure” is a strong word, but the authors are confident that it has been achieved ...
4-15-2019 drug pill pricing weigh endpts tile

Gene therapy could be revolutionary—if we can figure out how to pay for it

Dozens of revolutionary gene therapies that mend faulty strands of DNA are on their way, bringing the power to eliminate ...
sog

Can CAR-T cell therapy tackle solid tumors too?

Last summer, [Cecelia] Barron’s cancer went from stage 2 to stage 3 oligodendroglioma. Behnam Badie, her surgeon at City of Hope, ...
3-31-2019 thals patient

Gene therapy for beta-thalassemia blood disorder one step closer to European approval

The first gene therapy to treat a rare blood disorder is one step closer to approval [March 29] following a ...
gene

Why gene therapy’s potential is attracting intense interest from Big Pharma

Big Pharma has been struggling for years to produce drugs that are major breakthroughs in medicine. For these companies, gene ...
genome editing ria

Viewpoint: Call for moratorium on heritable gene editing reminds us of gene therapy’s troubled early days

A group of influential scientists have urged that clinical use of CRISPR be put on hold in the wake of ...
GEN

Inside the quest to use gene therapy to cure a form of congenital blindness

[I]nherited retinal disease affects thousands of people around the world—people who are born with poor vision and then slowly lose ...
alzheimers

Alzheimer’s disease targeted with gene therapy trial

There’s a test for Alzheimer’s risk that genetic counselors don’t like to talk about. It’s not that they’re hiding the ...
2-26-2019 alzheimers trial bathe brains genes

Gene therapy could offer ‘one-time genetic tune-up’ for Alzheimer’s patients

No one knows for certain what causes Alzheimer’s disease. But one fact about the condition has gained nearly irrefutable status ...
closeup of doctors hand holding ct brainscan

Gene from controversial CRISPR baby experiment could deliver new stroke treatment

A widely criticized experiment last year saw a researcher in China delete a gene in twin girls at the embryonic ...
2-18-2019 jim wilson mini e x

Pioneering ‘refugee’ from gene therapy’s darker days ‘kept the faith’

Gene therapy, now a pillar of biotechnology with the potential to cure deadly diseases, was once a cautionary tale of ...
2-19-2019 angelman clinic feature

Rare brain disorder—Angelman syndrome—could be treated with gene therapy in the womb

Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains ...
2-18-2019 british

First human test for gene therapy targeting most common cause of blindness

A woman from Oxford has become the first person in the world to have gene therapy to try to halt ...
genetherapy x

First gene-editing experiments on adults suffering from rare metabolic disorders offer promising early results

In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results ...
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Chinese scientists aren’t keeping tabs on experimental gene therapy patients, report says

Gene therapies are very much at their preliminary stages of development, so it would make sense to keep tabs on ...
sickle

Can gene therapy offer a cure for sickle-cell disease?

[I]n November, six months after [21-year-old Manny] Johnson became the first patient to receive an experimental therapy aimed at curing ...
IMG

Inside a couple’s quest to pay for an experimental gene therapy to save their children

“We need your help, we really do,” Gary [Landsman] says, his voice breaking. The Landsmans’ two sons—Benny, then 18 months, ...
Gene therapy for autism? Rare Angelman syndrome could answer key questions

Gene therapy for autism? Rare Angelman syndrome could answer key questions

[T]he success of genetic therapies [for autism] is not guaranteed. Even if disrupted genes can be replaced, repaired or otherwise ...
SOG

CAR-T cell therapy and the promise of immune cells engineered to fight cancer

The National Cancer Institute estimates that more than 1.7 million people will be diagnosed with cancer this year. To cope ...
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Can we afford gene therapy’s million-dollar price tags?

[4-year-old] Caspian was born with a rare, inherited eye disorder called Leber congenital amaurosis, which results in the progressive deterioration ...
nobel

Were ‘crucial contributors’ snubbed in awarding Nobel prize for cancer immunotherapy?

[I]t’s rare that Nobel announcements don’t produce grumblings about who was left out, and this year was no exception. At ...
FDA

Seeking a new path for FDA regulation of revolutionary medical treatments

Researchers are developing therapies that could permanently alter a patient’s genes... . The early results are promising. Patients would live ...
gene therapy

Bringing us closer to ‘once-in-a-lifetime’ hemophilia treatment with experimental gene therapies

Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After ...
gene therapy

Mainstream medicine? US health officials want to ease restrictions on gene therapy experiments

U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly ...
French Anderson AP x

Examining the legacy of W. French Anderson—’father of gene therapy’ and convicted child molester

[Dr. W. French] Anderson has been hailed as the father of gene therapy and was honored at George H.W. Bush’s ...