Ronald.L..Lahti@f8.n10.z1.fidonet.org (Ronald L. Lahti) (01/05/90)
Index Number: 6141
I must admit that this has been one of the most informative
conferences I have brought onto this machine! My fiancee is
actively trying to learn this computerized world of mine. She has a
disease called "Familia Spastic Paraplegia" (don't quote me on the
spelling). What I know of it, the disease is reasonably rare,
hereditary, and primarily affects women. Most of her family has
been afflicted with it. The disease eventually causes a loss of
speech and use of the lower extremities.
I am VERY interested in learning more about the condition, but
about all I get from the "SPECIALISTS'" is what I typed above. If
anyone out there knows more about it...ie., CAUSE and STUDIES, I
would appreciate hearing from you. BTW, her name is Schelia
Segroves (Lahti), and you will be hearing from her as she gets her
nerve up!! My compliments to all!
Blessings for the New Decade...
Ron
# Origin: [The Hawk's Nest - LaQuinta, CA] (8:911/302)
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Internet: Ronald.L..Lahti@f8.n10.z1.fidonet.orgRon.Rothenberg@f460.n101.z1.fidonet.org (Ron Rothenberg) (01/17/90)
Index Number: 6303
RL> Our lawyer found the information VERY useful! How do we
RL> contact the NORD group? Is this an on-line database or
RL> something? I am very interested in this and we would like to
RL> join very much. If that report was any example of the work
Hi Ron,
NORD is an organization and one of their services is the on-line
database. They also do lobbying and support, etc. Here's some of
their materials about their work and their services. They might even
be able to get you together with other people with the same disorder.
Living with any illness, disability or disorder is easier if you know
others are with you in the same boat for support, advice and mutual
assistance.
Their on-line database is available through Compuserve. If you're a
subscriber, just type GO NORD.
-rsr-
NORD---CREATED TO HELP
The National Organization for Rare Disorders (NORD) began in 1980 as an
informal coalition of small voluntary health agencies and individuals affected
by orphan diseases, to work toward passage of the Orphan Drug Act. Incorporated
in 1983, NORD has monitored the Orphan Drug Act to ensure its implementation to
the fullest extent possible. In addition, NORD has been called upon to address
the common needs and concerns of nearly twenty million Americans whose lives
are touched by Orphan Diseases.
ORPHAN DISEASES
A disease which affects fewer than 200,000 Americans is deemed an "Orphan
Disease." There are more than 5,000 such afflictions which strike people of
all ages, all races, and all ethnic backgrounds. Many are genetic, for
example: Tay Sach's disease, Huntington's disease, Cystic Fibrosis. Others
are acquired like AIDS, Legionnaire's disease, etc. A number of "orphans" fall
within the realm of wide spread health conditions such as heart disease and
cancer. For example, cancer will strike an estimated one out of every four
Americans, but only a few subtypes of cancer will affect more than 200,000
people at any one time. Thus, many people with cancer face the same problems
as people with lesser known ailments.
THE ORPHAN DRUG ACT
The limited number of Americans disabled by each orphan disease represents an
unprofitable market to developers of new orphan therapies (orphan drugs,
devices, biologics or antibiotics). To alleviate this serious health problem,
the Orphan Drug Act was passed into federal law on January 4, 1983. This law
provides economic incentives to pharmaceutical manufacturers who develop and
market orphan drugs. It also authorizes research funds for scientific
investigations into new orphan therapies. The law has yet to address
development of orphan devices. These products--like pacemakers used for
treatment of heart disease--are medically necessary to prevent, diagnose or
treat certain low incidence conditions.
THE MISSION OF NORD
NORD's mission is to encourage increased identification, control and cure of
orphan diseases through the following programs:
* Education: NORD educates the public and professionals so they may more
readily understand and alleviate rare disorders. The organization acts as a
clearinghouse for current information about more than 5,000 orphan diseases.
* Service: Through NORD's "Networking" program, NORD links people with the
same orphan disorder together for mutual support. In many instances, only a
handful of people in the nation may have an illness, and no voluntary agency or
support group may yet exist for that condition. NORD nurtures small support
groups through its technical assistance program, ensuring that new support
groups and voluntary agencies will evolve to serve the needs of each disease
population.
* Research: NORD encourages and supports research on understudied orphan
diseases, creating hope for millions whose disorders are presently hopeless or
untreatable.
ORGANIZATION OF NORD
NORD is governed by a Board of Directors composed of major leaders of national
non-profit health agencies, and individuals with rare disorders. The NORD
Research Advisory Council (NRAC) is comprised of outstanding leaders from
business, government, and academia who are determined to help raise funds which
will be used to further scientific research on rare disorders. It is their aim
to bring the fruits of scientific discoveries out of the laboratories and into
the home of patients who need them. NORD is fortunate to benefit from the
voluntary services of a prestigious Medical Advisory Board (MAB). Members of
this Board are expert medical professionals in many fields of basic and
clinical research. The MAB offers advice on important scientific issues and
will serve as a peer review group for evaluating requests for funding submitted
to the NORD Research Grant program.
NORD SERVICES
NORD Services offers the following:
* Rare Disease Database--This is a keyword searchable database with information
on hundreds of disorders. Each entry includes synonyms, a general discussion
of the disease, symptoms, etiology, affected population, related disorders
(where applicable), therapies (both standard and investigational), and
resources for further information concerning the disease.
* Newsletters--The newsletters of several voluntary health agencies are
available in this section, allowing readers to browse through a variety of
fascinating articles concerning specific types of disorders. However, many of
the articles will be of interest to the general public. News about research
projects on the cutting edge of science is available here.
* Prevalent Health Conditions/Concerns--Articles of general interest to the
public can be found in this section. Most are publications produced by the
Food and Drug Administration (FDA), Centers for Disease Control (CDC), National
Institutes of Health, etc. The articles tend to focus on more prevalent health
conditions or specific health topics which are newsworthy.
* Orphan Drug Database--This database is searchable by disease name with
information on those drugs and products designated as "orphans" by FDA. Entries
give both the generic and brand (where applicable) name of the drugs, the
disease and/or condition they are to be used to treat or diagnose, the
manufacturer and information about clinical trials. It will be updated as new
information becomes available.
ORPHAN DISEASE UPDATE
The following is a condensed version of NORD's newsletter. ORPHAN DISEASE
UPDATE is a vehicle for communication about rare disorders and is mailed
throughout the world. All views expressed herein are strictly those of the
authors and should not be construed to represent opinions or policies of NORD.
Subscription to the ORPHAN DISEASE UPDATE is included as part of NORD's
membership fee. For more information, please contact:
National Organization for Rare Disorders
P.O. Box 8923
New Fairfield, CT 06812
(203) 746-6518
WHAT IS NORD
Some of us have heard the story about the three blindfolded men who were asked
to touch an elephant and then describe the object they were touching. Each
described a different object, and none guessed that he was touching part of an
elephant. Asked to define NORD, we have found that members offer a wide
variety of descriptions, each according to the part of NORD which has touched
their lives. Very few understand or can visualize the entire elephant.
What is NORD? Begun as an informal coalition of national voluntary health
agencies during the late 1970's, the groups united around only one issue: the
unavailability of orphan drugs to patients with rare diseases. Orphan drugs
are therapies which are used to treat rare disorders, but because they were
unprofitable, they were not being manufactured by the pharmaceutical industry.
The informal coalition evolved into a grassroots effort to find a permanent
solution to this devastating problem.
The situation was serious. Researchers whose grants had expired were unable to
purchase raw ingredients on a continuing basis. Many of these therapies were
treatments for disorders which had no other known therapy. Patients suffered
needlessly, and some died. The pharmaceutical industry had tried to provide
some of these "service" drugs to the public, but the expenses of research
required for FDA approval were overwhelming when so many orphan drugs were in
need of development.
Finally, consumers and researchers testified before Congress, pleading for
immediate action. A small newspaper story appeared in the Los Angeles Times
about the testimony, and Jack Klugman's brother, Maurice, read that story. He
brought it to Jack's attention, and an episode of the NBC-TV show, Quincy,
M.E., was broadcast in 1981 about the plight of people with orphan diseases.
As a result of the Quincy show, thousands of people contacted members of the
informal coalition. Some asked how they could help, but others wrote about
their loved ones who suffered from rare, chronic, and debilitating disorders.
They asked for information about each illness and referral to knowledgeable
clinics or voluntary health agencies. The task was huge; when we tried to find
information about these disorders we quickly realized how little information
was available and that much of the literature was outdated.
Subsequently, NORD was begun in a small plastic box filled with index cards
listing names of rare disorders and referral sources for people who inquired
about these illnesses. Without the help of the dedicated scientists on our
Medical Advisory Board and the voluntary agencies knowledgeable about their
disorders (or related ones), none of those thousands of questions could have
been answered. It was hard work done solely by volunteers until NORD
incorporated and enough funding was gathered to hire part-time staff.
When NORD formalized into a non-profit health agency, it was no longer simply a
coalition. It filled a void in America's health care system by providing
information, referal, advocacy, and encouragement of orphan disease research.
NORD was no longer concerned merely with orphan drugs, but with ALL issues
related to rare disorders, including the low level of research, the lack of
accurate information, the difficulties of obtaining benefits such as Social
Security, and services such as health care or rehabilitation. NORD is
convinced that something must be done about discriminatory priorities of
institutions and government agencies that feel the nation's resources must be
spent only on "major" health threats. Rare disorders are not a mere minor
annoyance!
During 1984 through 1986, the need for NORD's services continued to grow. A
small article about NORD in the January 1986 issue of McCall's Magazine
elicited thousands of inquiries over a period of weeks. Where a reliable
voluntary agency exists for a rare disorder, we can be assured that patients
referred to that agency will obtain expert information and a sorely needed
support network. However, more than half the inquiries received at the NORD
office ask about disorders which are so rare that no voluntary agency yet
exists to serve these populations. To meet these needs, NORD created two
important programs: A) a computerized rare disease database, accessible to the
public and professionals through CompuServe; and B) NORD's "networking" program
links together families with the same disorder when they provide written
permission of release of their name. We hope this program will encourage
development of new support groups resulting in formation of more voluntary
agencies for even the most rare disorders.
In retrospect, the evolution of NORD seems natural, but the work quickly became
overwhelming. During the summer of 1985, NORD's Board of Directors decided to
open a new office and hire full-time staff. An imminent merger with the
National Orphan Drug and Device Foundation will expand NORD's programs again by
adding direct funding of scientific research on understudied disorders to
NORD's agenda. The merger will lend the efforts of the Foundation's
prestigious Board to NORD's vital programs.
Through the years, NORD has remained keenly aware of the necessity to help the
estimated 20 million Americans who have one of over 5,000 rare disorders.
While there has never been a lack of willingness, there has often been a lack
of funds to meet those needs.
To this date, NORD receives no government support and our programs rely solely
on public donations. We know there is much to be done, and we pray with your
help that we will have the resources to reach our goal of alleviating or curing
all of these dreadful illnesses. As one inquirer wrote at the end of a lengthy
letter, "I know that there is no cure for my disorder, but thank you for giving
me hope."
... Time wounds all heels.
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Internet: Ron.Rothenberg@f460.n101.z1.fidonet.orgRon.Rothenberg@f460.n101.z1.fidonet.org (Ron Rothenberg) (01/17/90)
Index Number: 6304
SL> totaly alone with this thing, but thru this board I've found
SL> that I'm not alone anymore, my other half really encourages me,
Hi Schelia,
Always glad to help. Here's the networking permission form from NORD.
When they receive the form, they will send you the names and addresses
of other people who are diagnosed with the same disorder and vice
versa. Then you really won't be alone. Best wishes and type at you
soon,
Ron
"NETWORKING" THROUGH THE NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD)
A disease which affects fewer than 200,000 Americans is deemed an "Orphan
Disease." There are more than 5,000 such afflictions which strike people of
all ages, all races, and all ethnic backgrounds. Many are genetic, for
example: Tay Sachs, Huntington's Disease, Cystic Fibrosis. Others are
acquired like AIDS, Legionnaires' Disease, etc. A number of "orphans" fall
within the realm of wide spread health conditions such as heart disease and
cancer. For example, cancer will strike an estimated one out of every four
Americans, but only a few subtypes of cancer will affect more than 200,000
people at any one time. Thus, many people with cancer face the same problems
as people with lesser known ailments.
Through NORD's "Networking" program, NORD links people with the same orphan
disorder together for mutual support. In many instances, only a handful of
people in the nation may have an illness, and no voluntary agency or support
group may yet exist for that condition.
If you wish to be put in contact with other people (or families) concerned
about the disorder you inquired about, please print out the form on the next
page and mail it to the address below. NORD will not add the names of third
parties (i.e., teachers, social workers, etc.) to the NORD Networking Program.
Therefore, the patient or his immediate family must sign this form in order to
participate in this program. This form cannot be sent to NORD via E-mail; it
must be sent through the Postal Service.
National Organization for Rare Disorders, Inc. (NORD)
P.O. Box 8923
New Fairfield, CT 06812
(203) 746-6518
The National Organization for Rare Disorders (NORD) keeps names and addresses
confidential unless a voluntary agency exists for the specific disorder, or a
person (or parent) provides written permission for NORD to send their name to
other people (or families) with the same disorder. The NORD Networking Program
is meant to encourage formation of mutual self-help support groups concerned
about a single rare disorder.
PLEASE NOTE: DO NOT SIGN THIS FORM IF YOU DO NOT INTEND TO CORRESPOND WITH
PEOPLE WHO HAVE YOUR SPECIFIC DISABILITY. IF YOU DO NOT ANSWER THEIR LETTERS,
YOU MAY UNNECESSARILY DISAPPOINT THEM.
WRITTEN PERMISSION FORM FOR NORD'S "NETWORKING" PROGRAM
NAME:
ADDRESS:
CITY:
STATE:
ZIP:
TELEPHONE NUMBER:
NAME OF RARE DISORDER:*
CIRCLE YOUR RELATIONSHIP TO THE PERSON WITH THE RARE DISORDER:
PATIENT PARENT SPOUSE
RELATIVE FRIEND
OTHER (SPECIFY)
* PLEASE NOTE: YOU MUST PROVIDE A NAME FOR YOUR DISEASE OR CONDITION. NORD
CANNOT DIAGNOSE PATIENTS NOR CAN WE MATCH NAMES OF PATIENTS ACCORDING TO
SYMPTOMS.
... Time wounds all heels.
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