Preempting severe genetic disease
Around 2% of births come with a genetic disease, often with devastating impact. While some appear de novo (either from germplasm dominant mutations or from chromosoma…
The new Humira?
It’s not that often that you see industry-sponsored head-to-head studies of one biologic vs. another, but Abbvie took the plunge comparing risankizumab (Skyrizi) to ustekinumab (Stela…
A Chinese study uncovers a new use for an old drug
Bleeding in the small intestine is often due to vascular malformations and is difficult to diagnose and manage because 1) the area is hard to reach w…
How the US taxpayer saved 25M+ people
A short piece celebrating the 20th anniversary of PEPFAR, a program that few outside of the field of Global Health are aware of. The world is not always nasty and…
Hypertension?!
Antihypertensive drugs are so 80s and 90s (well, except for pulmonary hypertension) to the point that I cannot recall when I last read research in the NEJM about a new blood pressure dr…
A psychedelic for refractory depression
While major depression is awful, treatment-resistant depression (as defined not responding to at least two different courses of therapy) is worse with sufferers…
After the $9.7B acquisition of The Medicines Company two and a half years ago, Novartis is eager to demonstrate value with a successful launch of the primary target of the deal, Leqvio. This launch is…
Gene therapy in beta-thalassemia and sickle cell anemia
Beta-thalassemia and sickle cell anemia are common genetic diseases of hemoglobin (Hb) which manifest themselves in the former through transfusi…
Incremental progress in the fight to treat heart failure
After a decade or more of relative quiescence in the 2000s, a volley of new therapies have come to the forefront in the pages of the NEJM: sacu…
Cardiovascular health – go big or go narrow?
A follow up on the development of evinacumab (Regeneron), an inhibitor of ANGPLT3 (see this opinionated take from 2017), confirming the safety and LDL red…
Small but real progress in ALS:
Despite enormous public attention and significant effort, ALS remains a disease for which the development of new therapies has been challenging. Animal models showed po…
The loneliness of the patent-less drug
Colchicine is a very old drug commonly used in gout with an anti-inflammatory mechanism of action that is not well defined. It is a generic (although in the US, …
TAVR for all?
TAVR has become the standard of care for patients who need an aortic value replacement but are at high/medium surgical risk. But what about those at low-risk? Two studies answer that qu…
Target assessment with genetic polymorphisms
Please bear with me for a long (but interesting!) story. Bempedoic acid (Esperion) is an inhibitor of ATP citrate lyase (ACLY), an enzyme in the cholestero…
Peanut medicine that won’t cost peanuts
Allergy to peanuts is a major issue and though prevention is now possible in infants there is a huge population for whom actual survival is connected to vigilan…
Primary care organizations are better ACOs when it comes to achieving savings
Initial results from the Medicare Shared Savings Program ACO have been disappointing pointing to small to negligible net e…
Are we nearing an asymptote with implantable cardiac pumps?
Severe heart failure is common, and spare hearts for transplant are rare, which has led to the development of implantable mechanical alterna…
A-fib in heart failure – time to be aggressive
Over the last 15 years, there has been a growing body of evidence for the effectiveness of catheter ablation to treat atrial fibrillation (a-fib), a cond…
Between the very common and the very rare – An opinionated take on NEJM highlights for October 2017
Another tool for prevention in cardiovascular disease
Taking aspirin daily has been standard of ca…
Taking stock: two decades of progress in heart failure:
Here comes a clever study using existing clinical trial data to assess progress in standard of care over time for heart failure. For each trial,…
A hammer finds new nails (which happen to be eyeballs)
The insulin growth factor receptor 1 (IGF-1R) was once upon a time a popular cancer target pursued by multiple biopharmas each with their own hum…
Calendaring care
The length of our sidereal year is an accident – we happen to be circling a G2 star from which the habitable zone where free surface liquid water can exist lies at around 150,000,000 …
Working Paper
Summary
Drug companies are naturally incentivized to price their drugs under assumptions of optimal clinical value, i.e. as high as possible. Payers react to this by setting stri…
Gene therapy for sickle cell disease
Typical diseases targeted by gene therapy are those for which there is a defect that prevents the production of a functional protein needed for normal life; remedi…
Surgical volume and referral for surgery:
The impact of surgical volume on outcomes has been well documented, but is it top of mind with physicians referring patients to surgery? Readers of the Journa…
Systems biology finally gets real: an unexpected use for a diabetes drug
Chronic Myelogenous Leukemia (CML) has been the poster child first for a disease with a precise genetic cause (the Philadelphia…
Interventionalist treatment for stroke:
In the 80s and 90s, treatment of myocardial infraction was greatly advanced by the introduction of systemic clot busting drugs (t-PA and others); further advanc…
The rise, fall, and rebirth of the Chinese healthcare system
A fascinating account of the evolution of the Chinese healthcare system which almost seems to be an upside-down picture of the rest of the …
A promising agent for Crohn’s Disease, a miserable illness
Crohn’s is an inflammatory bowel disease that is notoriously unpredictable; flares can affect any part of the digestive tract and lead to gra…
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