understanding and filtering information
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Understanding and Filtering Information presented by Tom Rando at PPMD's 2010 Annual Connect ConferenceTRANSCRIPT
Understanding and Filtering the Information
Thomas A. Rando, MD, PhD
Department of Neurology and Neurological Sciences Stanford University School of Medicine
VA Palo Alto Health Care System
CONNECT Conference Parent Project Muscular Dystrophy
Denver, CO June 24, 2010
The Information Revolution in DMD 1980 1990 2000 2010
INFORMATION:
Quantity
Access
Molecular diagnostics
Experimental Therapeutics
Clinical Trials
Email, Listservs
Internet Websites
Social Networking
Minimal new information, Limited access
Much new information, Limited access
Much more information, Unlimited access
You
Doctors Agencies
Pharmaceutical Companies
Friends, Relatives
Scientists
Understanding the Diagnosis: One Step at a Time
- Clinical status - Short term: signs and symptoms - Long terms: prognosis
- Laboratory Tests - Short term: supporting the diagnosis, other testing? - Long term: genetics, carrier status
- Therapeutics
- Short term: physical therapy, steroids? - Long term: other medical/surgical, clinical trials
What You Should Expect from Your Doctor
- A clear explanation of the clinical diagnosis and prognosis;
What You Should Expect from Your Doctor
- A clear explanation of the clinical diagnosis and prognosis;
- A general explanation of what the tests showed;
What You Should Expect from Your Doctor
- A clear explanation of the clinical diagnosis and prognosis;
- A general explanation of what the tests showed;
- Time to answer your questions of each of those;
What You Should Expect from Your Doctor
- A clear explanation of the clinical diagnosis and prognosis;
- A general explanation of what the tests showed;
- Time to answer your questions of each of those;
- A near-term plan for further tests and any therapy;
What You Should Expect from Your Doctor
- A clear explanation of the clinical diagnosis and prognosis;
- A general explanation of what the tests showed;
- Time to answer your questions of each of those;
- A near-term plan for further tests and any therapy;
- Suggestions of where to go for further information;
- A detailed understanding of the genetics
What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)
- A detailed understanding of the genetics
- A specialist’s understanding of the test results
What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)
- A detailed understanding of the genetics
- A specialist’s understanding of the test results
- A familiarity with the clinical trials that are underway or planned
What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)
Where to Obtain the Best Information
Parent Project Muscular Dystrophy:
http://www.parentprojectmd.org
Muscular Dystrophy Association:
http://www.mdausa.org
Muscular Dystrophy Campaign:
http://www.muscular-dystrophy.org
Filtering the “Information”
Press Releases
Most accessible, Fewest details
Least accessible, Most details
Research Announcements
Journal Articles
Interpreting Press Releases
Stem cell hope for Duchenne muscular dystrophy Posted on July 17, 2008 by speakingofresearch
Duchenne muscular dystrophy (DMD) is an inherited disease that affects about one in every 4,000 males born in the USA. It is caused by mutations in the DMD gene that lead to the protein dystrophin being either absent or faulty, which leads to muscle cell death, progressive muscle wasting and early death, with few patients surviving beyond their 40th birthday.
In recent years scientists have been investigating the possibility of transplanting healthy stem cells into the muscle of patients in order to replace the lost muscle cells and halt the progression of the disease, but it has proved difficult to identify muscle precursor cells that can both make new muscle cells and persist as a pool of precursor cells in the patient, the latter is an important consideration if repeated transplants are to be avoided since muscle cells wear out and need to be replaced. A paper published in this weeks issue of the scientific journal Cell by scientists at Harvard University is an important step towards developing a means to screen for the right cells and use them to treat DMD
Understanding Research Announcements
Assessing Journal Articles
Understanding the “Information”
Press Releases
Most accessible, Fewest details
Least accessible, Most details
Research Announcements
Journal Articles
Information Overload
- Don’t try to become a neurologist/geneticist/cell biologist
Information Overload
- Don’t try to become a neurologist/geneticist/cell biologist
- Understand the process from the laboratory to the clinic
Information Overload
- Don’t try to become a neurologist/geneticist/cell biologist
- Understand the process from the laboratory to the clinic
- Focus on issues related to current treatments (e.g. steroids)
Information Overload
- Don’t try to become a neurologist/geneticist/cell biologist
- Understand the process from the laboratory to the clinic
- Focus on issues related to current treatments (e.g. steroids)
- Find the sources you trust for information on long term issues
Information Overload
Information is valuable.
Time is priceless.