Mystery Solved: Which Patients Are Good Candidates For Acute Inpatient Rehabilitation?
Occupational Therapy Environment, Saint Luke's Hospital, WA
For most physicians who practice inpatient medicine, acute inpatient rehabilitation facilities are mysterious places with inscrutable admissions criteria. This is partly because physical medicine and rehabilitation (PM&R) has done the poorest job of public relations of any single medical specialty (Does anyone know what we do?), and also because rehab units have been in the cross hairs of federal funding cuts for decades. The restrictive CMS criteria for inpatient rehabilitation have resulted in contortionist attempts to practice our craft in an environmen...
Source: Better Health - June 16, 2014 Category: American Health Authors: Dr. Val Jones Tags: Health Policy Health Tips 60% Rule Acute Inpatient Rehabilitation Acute Rehab Admissions Coordinator Admissions Criteria Admissions Guidance ARF ARU Case Manager CMS Hospitalists Physical Medicine And Rehabilitation PM&R Rule Source Type: blogs
How 23andMe got in trouble
Open sesame?
Lukas Hartmann shares a great, detailed story (My deadly genetic disease was just a bug) of what happens when a self-described nerd is “confronted with a life threatening situation,” in this case a message about him from 23andMe that read:
“Has two mutations linked to limb-girdle muscular dystrophy. A person with two of these mutations typically has limb-girdle muscular dystrophy.”
Turns out this was the result of an error by 23andMe’s computer system. Now I’m all for patients having control over their lab results and getting away from the paternalistic idea that all results...
Source: Health Business Blog - February 10, 2014 Category: Health Managers Authors: David Williams Tags: e-health Research Source Type: blogs
Embryos with Genetic Disease Destroyed for Their Stem Cells, Cell Lines Added to NIH Registry
King's College in London is proud that they have provided 16 new embryonic stem cell lines to the United States' National Institutes of Health (NIH) registry of stem cell lines eligible for federal funding. On the King's College website they explain that they have created these stem cell lines with a variety of genetic disease for US researchers to work on:Scientists from Kings College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the ...
Source: Mary Meets Dolly - November 26, 2013 Category: Geneticists and Genetics Commentators Tags: Stem cells, Embryonic Source Type: blogs
New Disease Targets for Old Drugs; Another Big Data Initiative
Genomic sciences and the growing interest in precision medicine are having a variety of different effects on healthcare delivery. For example, patients are now being triaged into special clinics focusing on the most aggressive tumor types or tumors of unknown primary (see: New Clinic for High-Risk Prostate Cancer Patients at the University of Michigan; Specialized Clinic Opens for Patients with Cancer of Unknown Primary). We are also now seeing the emergence of hospital executives with portfolio emphasizing precision medicine (see: Do We Need Vice Deans/Vice Presidents for Precision Medicine?; A Closer L...
Source: Lab Soft News - November 21, 2013 Category: Pathologists Authors: Bruce Friedman Tags: Healthcare Information Technology Hospital Executive Management Hospitals and Healthcare Delivery Laboratory Industry Trends Medical Research Pathology Informatics Pharmaceutical Industry Source Type: blogs
Sarepta's Approval Woes
I briefly mentioned Sarepta and etiplirsen, their proposed therapy for Duchenne muscular dystrophy (DMD) in September. In that post, I made reference to the "delirious fun of investing in biotech". Well, the company recently got some regulatory news that illustrates that point even more clearly. The FDA told Sarepta that it would not get accelerated approval for the drug, and that sent the stock into a mineshaft (and infuriated the DMD community, as you might well think).
Matthew Herper at Forbes has some good background on the story here Etiplirsen is one of these drugs aimed at a small market (one particular DMD mutatio...
Source: In the Pipeline - November 13, 2013 Category: Chemists Tags: Clinical Trials Source Type: blogs
A Wall Street Darling No More: FDA Sends Sarepta Plunging
One of the hottest biotech stories over the past year went ice cold today when Sarepta Therapeutics revealed the FDA decided it would be “premature” to proceed with paperwork for its eteplirsen drug to treat Duchenne muscular dystrophy, or DMD. The disclosure sent Sarepta shares plunging nearly 60 percent this morning and appears to have ended its run as a Wall Street darling, at least for now.
In explaining the setback, Sarepta was told the FDA believes there is “considerable doubt” about a proposed biomarker – an increased production of dystrophin, which is a protein that Duchenne patients are lacking. The agen...
Source: Pharmalot - November 12, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
Catalyst Pharma Fights Greed Charges Over An Orphan Drug
For the past month, shares in a small developer of novel drugs called Catalyst Pharmaceutical Partners have given up roughly half their value amid an unusual public relations war with a privately held drugmaker over a potentially lucrative market for an orphan medication. But the tale has some twists that include a decommissioned nuclear reactor and free dosing for existing patients.
Here is the situation: Catalyst licensed a drug from Biomarin Pharmaceuticals called Firdapse to treatment Lambert-Eaton Myasthenic Syndrome, or LEMS, a rare autoimmune disorder that is characterized by muscle weakness and often associated wit...
Source: Pharmalot - November 6, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
Catalyst Pharmaceuticals And Their Business Plan
The orphan-drug model is a popular one in the biopharma business these days. But like every other style of business, it has something-for-nothing artists waiting around it. Take a look at this article by Adam Feuerstein on Catalyst Pharmaceuticals, and see what category you think they belong in.
They're developing a compound called Firdapse for Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuromuscular disorder. It's caused by an autoimmune response to one set of voltage-gated calcium channels in the peripheral nervous system. Right now, the treatments for the condition that seem to provide much benefit are intravenou...
Source: In the Pipeline - October 21, 2013 Category: Chemists Tags: Regulatory Affairs Source Type: blogs
Pharmalot... Pharmalittle... The Weekend Nears
And so, another working week will soon draw to a close. This is, as you know, our treasured signal to daydream about weekend plans. Today, however, the daydreaming begins early because we are officially off today, although we have obviously stopped for a few moments to conduct some business here. In any event, our agenda is filled with excitement, such as celebrating a birthday with one of the Pharmalot ancestors and taking Mrs. Pharmalot to yet another installment in our 'Let's-See-Them-Before-They-Die' concert series. But what about you? Anything interesting planned? How about catching up with someone special or reading ...
Source: Pharmalot - October 11, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
A Glaxo Drug For A Rare Type Of Muscular Dystrophy Is A Flop
In a blow to GlaxoSmithKline and a fledgling partner, the results of a Phase III study for a muscular dystrophy drug failed to meet its primary endpoint, a surprising outcome that caused caused shares in Prosensa Holding to plunge and prompted Wall Street to cautiously boost the outlook for a rival drugmaker, Sarepta Therapeutics.
The study was testing drisapersen for Duchenne muscular dystrophy, or DMD, a rare form of the disorder, in 186 boys for nearly a year and found there was no statistical difference in their performance in six-minute walking distance test compared with those given a placebo. There was also no stati...
Source: Pharmalot - September 20, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
A Glaxo Drug For A Type Of Muscular Dystrophy Is A Flop
In a blow to GlaxoSmithKline and a fledgling partner, the results of a Phase III study for a muscular dystrophy drug failed to meet its primary endpoint, a surprising outcome that caused caused shares in Prosensa Holding to plunge and prompted Wall Street to cautiously boost the outlook for a rival drugmaker, Sarepta Therapeutics.
The study was testing drisapersen for Duchenne muscular dystrophy, or DMD, a rare form of the disorder, in 186 boys for nearly a year and found there was no statistical difference in their performance in six-minute walking distance test compared with those given a placebo. There was also no stati...
Source: Pharmalot - September 20, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
A Glaxo Drug For Muscular Dystrophy Flops In Late-Stage Trial
In a blow to GlaxoSmithKline and a fledgling partner, the results of a Phase III study for a muscular dystrophy drug failed to meet its primary endpoint, a surprising outcome that caused caused shares in Prosensa Holding to plunge and prompted Wall Street to cautiously boost the outlook for a rival drugmaker, Sarepta Therapeutics.
The study was testing drisapersen for Duchenne muscular dystrophy, or DMD, a rare form of the disorder, in 186 boys for nearly a year and found there was no statistical difference in their performance in six-minute walking distance test compared with those given a placebo. There was also no stati...
Source: Pharmalot - September 20, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs
Prosensa: One Duchenne Therapy Down
In the post here the other day about Duchenne Muscular Dystrophy (DMD) I mentioned two other companies that are looking at transcriptional approaches: Prosensa (with GSK) and Sarepta. They've got antisense-driven exon-skipping mechanisms, rather than PTC's direct read-through one.
Well, Sarepta still does, anyway. Prosensa and GSK just announced clinical data on their agent, drisapersen, and it appears to have missed completely. The primary endpoint was a pretty direct one, total distance walked over six minutes, and they didn't make statistical significance versus placebo. This was over 48 weeks of treatment, and none of...
Source: In the Pipeline - September 20, 2013 Category: Chemists Tags: Business and Markets Source Type: blogs
The Arguing Over PTC124 and Duchenne Muscular Dystrophy
This article at Nature Biotechnology does an excellent job explaining the details.
Premature "stop" codons in the DNA of DMD patients, particularly in the dystrophin gene, are widely thought to be one of the underlying problems in the disease. (The same mechanism is believed to operate in many other genetic-mutation-driven conditions as well. Ataluren is supposed to promote "read-through" of these to allow the needed protein to be produced anyway. That's not a crazy idea at all - there's been a lot of thought about ways to do that, and several aminoglycoside antibiotics have been shown to work through that mechanism. Of t...
Source: In the Pipeline - September 18, 2013 Category: Chemists Tags: Business and Markets Source Type: blogs
The Down Syndrome Conundrum
Consider, if you will , the following statement:"If muscular dystrophy were completely cured, the world would lose something from the absence of that culture" Or, perhaps:"If celiac disease were completely cured, the world would lose something from the absence of that culture"Would you agree with either of these?How about this one:"If Down syndrome were completely cured, the world would lose something from the absence of that culture" That's the potential of promising new research being done at the University of Massachusetts Medical School, which claims that "it may be possible to switch off the genetic material responsib...
Source: InsureBlog - August 12, 2013 Category: Medical Lawyers and Insurers Source Type: blogs
