FDA is taking on tampons and ‘misleading’ ads starring Serena Williams
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Category: rare diseases
STAT+: A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
After his son Michael was diagnosed with a devastating, ultra-rare disease in 2019, Terry Pirovolakis…
STAT+: Gene therapy pioneer Jim Wilson believes he can commercialize rare-disease drugs in a tough climate
If you’d told Jim Wilson three years ago that he’d be stepping down as director…
STAT+: A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases?
Will it work? It was the question everyone around Susannah Rosen kept asking. Since infancy,…
STAT+: ‘Jerking families around’: Canceled Roche rare disease trial devastates parents, angers researchers
Roche, the Swiss pharmaceutical giant, earlier this year abruptly cancelled an international clinical trial of…
Listen: The long journey to make malaria vaccines, and Sarepta’s ties with patient advocates
Why has it taken so long for the world to get malaria vaccines? What relationship…
STAT+: Jim Wilson, gene therapy pioneer, departs Penn to set up new companies
Jim Wilson, a leading gene therapy researcher who has persisted through the field’s ups and…
STAT+: With new data, RegenxBio advances plan for Duchenne gene therapy
Regenxbio said Thursday that its experimental gene therapy for Duchenne muscular dystrophy was administered to…
STAT+: Parasite engineered to treat brain development disorder in mice shows potential in study
The human body works hard to prevent outside substances from entering the brain — which…
STAT+: Sarepta demanded Duchenne patient advocacy group censor video critical of the company
Sarepta Therapeutics demanded a prominent patient advocacy organization censor a video that contained pointed criticism…
Opinion: The rare pediatric disease voucher program creates new treatments. I have new data to prove it
The Food and Drug Administration’s rare pediatric disease priority review voucher program, which has been…
Can JAK inhibitors bolster checkpoint inhibitors?
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STAT+: Top FDA official Peter Marks overruled staff, review team to approve Sarepta gene therapy
For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official…
STAT+: FDA approves Sarepta’s Duchenne gene therapy for nearly all patients
The Food and Drug Administration on Thursday approved Elevidys, Sarepta’s gene therapy for Duchenne muscular…
Opinion: Readers respond to a plea for rare disease progress, vaccine-related injury, and more
First Opinion is STAT’s platform for interesting, illuminating, and maybe even provocative articles about the…
Listen: Duchenne gene therapy setback, Alzheimer’s drug endorsement & why a STAT reporter buys weed (for journalism)
On this week’s episode of “The Readout LOUD,” our STAT colleague Nicholas Florko joins us…
STAT+: Europe is set to shake up the way it assesses new medicines. Gene therapy makers are worried
LONDON — A major shakeup in how the benefits of medicines are assessed in Europe…
STAT+: Pfizer’s gene therapy for Duchenne muscular dystrophy fails in phase 3 trial
Pfizer said Wednesday afternoon that a closely watched gene therapy for Duchenne muscular dystrophy failed…
Opinion: Curing rare childhood diseases will falter unless Congress steps up
When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the…
STAT+: Agios drug reduces blood transfusions in patients with beta-thalassemia, study shows
Agios Pharmaceuticals said Monday that its drug called mitapivat reduced the need for blood transfusions…
Listen: An ASCO preview & another Duchenne trial failure
Which presentations are worth catching at ASCO this weekend? What is exon-skipping? And how do…
STAT+: Japanese conglomerate to buy Calliditas Therapeutics, maker of a kidney disease drug
LONDON — Seeking to build up its pharma business, the Japanese conglomerate Asahi Kasei is…
STAT+: Duchenne muscular dystrophy drug from Nippon Shinyaku fails in rare confirmatory trial
Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug, Viltepso, part…
STAT+: Biogen, Ionis drop ALS treatment after study failure
A plan to use new genetic techniques to treat non-genetic forms of ALS failed in…
STAT+: Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests
Two years ago, Yiwei Shi was searching desperately for someone to build a drug for…
STAT+: After its last deal fell through, a rare disease therapy gets a second chance with Shionogi
After seeing its last deal go bust in the face of regulatory scrutiny, Maze Therapeutics…
STAT+: Experimental gene therapy seems to alleviate skeletal defects tied to rare inherited disease, study shows
LONDON — In 2021, a team of Italian researchers reported that an experimental gene therapy…
STAT+: Why a British hospital, and not a drugmaker, is trying to get a rare disease therapy approved
LONDON — Claire Booth, a gene therapy researcher in London, had hoped that a biotech…
How resurrecting extinct species might impact medicine
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STAT+: Early data from rare-disease trial put spotlight on Moderna’s efforts to turn mRNA into medicines
LONDON — Moderna may be best known for its Covid-19 vaccine, but since its start,…
STAT+: Critics say U.K. decision on ALS drug could have ‘chilling effect’ on access to new genetic medicines
LONDON — Neurologists and patient advocates are up in arms over a policy decision by…
STAT+: U.K. health officials and researchers try to carve a path for more individualized genetic treatments
LONDON — Widespread genetic testing as well as the development of cutting-edge, customized genetic therapies…
STAT+: FDA approves first gene therapy for a fatal neuron disease in children
The Food and Drug Administration on Tuesday approved a gene therapy for metachromatic leukodystrophy, a…
STAT+: AstraZeneca picks up rare-disease focused Amolyt Pharma in $1 billion deal
LONDON — AstraZeneca said Thursday it would purchase the French firm Amolyt Pharma in a deal…
STAT+: He’s 19 months old and has one of the world’s rarest diseases. His parents are determined to find a cure.
SOMERVILLE — At 19 months old, Lucas Guo has never crawled, walked, or stood by…
The FDA isn’t afraid to yank a disappointing drug
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Texas Medicaid agrees to fully cover gene therapy for Afghan refugees’ infant
After a months-long fight with Texas Medicaid over coverage of a gene therapy, Afghan refugees…
STAT+: Obscure vertigo drug could bring rare hope for patients with devastating neuron disease
About a decade ago, Tatiana Bremova-Ertl’s graduate adviser was studying an obscure, 1950s-era French vertigo…
In partial reversal, Texas Medicaid greenlights first step in gene therapy for Afghan refugees’ infant son
The Pashai family of Dallas on Thursday got some good news, but not exactly the…
The Afghan father aided U.S. soldiers. Now Texas Medicaid won’t pay for his son’s gene therapy
On a sweltering day in August 2021, as the engines of a hulking C-17 cargo…
This is the TLDR of JPM Day 1
In honor of JPM Week, you’re reading a special edition of our biotech newsletter, The…
FDA approves AstraZeneca, Ionis treatment for rare nerve disease
The Food and Drug Administration approved a novel medicine for a rare and devastating nerve…
Morning Rounds: Why health care costs are nearly a fifth of the GDP
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BioMarin appeases an activist
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The legal pathway for experimental, ultra-rare disease treatments is rocky and expensive
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STAT+: ‘We’ve exhausted all avenues’: A small biotech may give up on its ultra-rare disease drug over frustration with FDA
Jamie Dubuque faces the future with a mixture of gratitude and trepidation. Her son, Declan,…
Morning Rounds: For kids with high BMI, task force not ready to recommend weight loss drugs
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A plot to solve the ‘whack-a-mole’ of drug discovery
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Gene therapy offered this 7-year-old freedom. The price: a grueling year
PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just…
Tome Biosciences goes public today in another biotech megaround
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