1. Services
for the Individual,
Family and Community
Updated February 2011

2. Dear Friends,
W hen we first learned that our daugh- This booklet also will give you an idea of
ter Abbey has a progressive muscle the many ways to get involved with MDA’s
disease, we knew that one of our great- mission. Our family has found that volun-
est assets for coping with the challenges teering at a fundraiser, getting a friend to
ahead would be our strength as a family. participate in a special event, lending our
voices to advocacy campaigns and other
But little did we know that we would find activities have helped us feel like we’re
an entirely new family to walk alongside doing all we can to further the search for
us as we faced fears, met new challenges cures and treatments.
and celebrated newfound joys. We’d like
to welcome you to what we hope will As we’ve learned how Abbey’s muscle dis-
become an extension of your family as ease will affect — but not limit — her life,
well: the Muscular Dystrophy Association. MDA has been right there with informa-
tion, medical care, unmatched support and
The journey that brought you to MDA may friendship. May you find similar refuge in
have been long, confusing and frustrating this community — and please don’t ever
as you sought answers or a diagnosis for hesitate to turn to your new family for help
yourself or a loved one. Whatever path and hope!
brought you here, please know that you’re
no longer alone: The entire MDA com- With love,
munity — staff, physicians, researchers,
sponsors, clinicians, donors, and families
and individuals — is here for you. Joel and Wendi Umali
Parents of Abbey Umali
In this booklet you’ll find information
2011 MDA National Goodwill Ambassador
about specific services that MDA offers,
and how to best utilize these many
resources, which can make a critical dif-
ference in understanding, managing and
living day-to-day with neuromuscular dis-
ease. MDA provides medical care, disease-
specific information, online and face-to-
face support, help obtaining and repairing
durable medical equipment, summer camp
for youngsters and — above all — a
world-class research program that some-
day will put an end to muscle diseases for
good.
2 MDA Services • ©2011 MDA

3. The Muscular Dystrophy Association is Here to Help
M DA is the gateway to information, n Connecting with others in your area
resources and specialized health with a similar diagnosis
care for individuals and families coping n Informational brochures and
with muscle disease. At more than 200 publications
local offices around the country, MDA’s n Support groups (in person, on the
compassionate and knowledgeable staff phone and online)
provides support and guidance. n Support and resources for caregivers
n Free flu shots
MDA’s comprehensive website (www.
n Presentations to your child’s school
mda.org) is another valuable source of
“We probably would not have survived information about neuromuscular disease, n Referrals to community-based
the last four years without the help of research news and MDA programs. resources
the Muscular Dystrophy Association.
n Advocacy
When we were introduced to them,
our lives changed. We were able to This booklet provides basic information n How you can join the fight against
network as parents and meet other about MDA services and programs. For neuromuscular disease
parents going through similar experi- information specific to you, contact your
ences. The staff at our local MDA n … and more.
office has been wonderful. They’ve local MDA office.
just accepted us as part MDA is here to help. If your local MDA
of their family. ” Turn to MDA with questions about: office can’t address a specific need, they’ll
n Specialized health care and referrals help you find other resources. Don’t hesi-
Robin Pennell, Virginia
Parent of a child affected by n Research advances in your disease tate to contact MDA for assistance and
limb-girdle muscular dystrophy n Durable medical equipment needs answers.
n Explaining your disease to family mem-
bers and others
MDA is Hope
concerned individuals and cooperating
What is MDA?
organizations. These programs include:
In 1950, a group of adults with neu-
romuscular diseases and parents of Research
children with these disorders joined MDA is among the foremost funders in
forces to form the Muscular Dystrophy the world of research into neuromuscular
Association. Recognized by the American diseases. MDA-funded scientists are at
Medical Association “for significant and the cutting edge of research into cell-
lasting contributions to the health and based therapies and testing of potential
welfare of humanity,” MDA raises funds treatments for the more than 40 diseases
to defeat more than 40 forms of neuro- under its umbrella. For the most current
muscular disease through programs of research news, go to mda.org/diseases
worldwide research, medical and support and click on a disease name.
services, professional and public health
Clinics (for more, see page 5)
education, and advocacy. The Association
At facilities in MDA’s nationwide network
provides services in the United States
of clinics, individuals receive diagnostic
and Puerto Rico through more than 200
and follow-up care from expert teams of
local offices.
muscle-disease specialists.
MDA’s programs are funded almost
entirely by the voluntary contributions of
3 MDA Services • ©2011 MDA

4. Support (for more, see page 8) Assistance with Medical Services
You are not alone. MDA is here every MDA will assist with payment for select
step of the way, providing resources and services through MDA clinics or local
guidance, assistance locating and repair- medical equipment providers. To maxi-
ing needed equipment, support groups, mize MDA resources, payment is made
summer camps for youngsters, online after private and public insurance and/or
“e-communities” and much more. other resources have paid. Please be sure
to contact the MDA office in your area
Information (for more, see page 9) before ordering services, if MDA payment
Knowledge is power. MDA conducts edu- is desired.
cational programs for individuals with
neuromuscular diseases, the general
public and medical professionals, includ-
ing publishing a wide variety of print and
audiovisual materials, and maintaining
three websites. MDA sponsors scientific
symposia and other professional meetings
to increase knowledge of neuromuscular
disease among medical professionals.
Advocacy (for more, see page 10)
MDA’s advocacy efforts are commit-
ted to making life better for people with
neuromuscular diseases by providing
“When our son was first diagnosed, we
didn’t know anything about muscular representation in matters of public policy
dystrophy other than we had watched and research advancement, nationally and
the Telethon since we were little kids.
internationally; and by facilitating active
Within 24 hours, we had contacted the
Muscular Dystrophy Association and involvement in these areas by the people
that was a fantastic move to make. it serves.
We learned throughout that there was
hope.”
Accessing MDA Services
Doriann Myers, Nevada
Parent of a child affected by Initial Diagnosis
Duchenne muscular dystrophy
The first step to access MDA services is
attending an MDA clinic to confirm your
diagnosis of one of the neuromuscular
diseases covered by MDA (see page 12 for
a complete list).
If the disease diagnosed is not covered by
MDA, referrals will be made to an appro-
priate health or community agency.
4 MDA Services • ©2011 MDA

5. MDA is Help
MDA has organized 10 of its elite clinics
The MDA Team into networks to support and speed clini-
MDA District offices are your headquarters cal trials of promising research. Clinics
for access to specialized health care at in the Duchenne Muscular Dystrophy
MDA clinics, as well as for local support Clinical Research Network are Washington
groups, summer camp, resource referral University in St. Louis; Boston Children’s
and more. Staff at your local MDA office Hospital; Nationwide Children’s Hospital in
can provide information, support and Columbus, Ohio; University of California
guidance. Davis in Sacramento, Calif.; and University
of Minnesota in Minneapolis.
In fact, MDA should be your first phone
call when you have nonmedical questions Clinics in the ALS Clinical Research
about living with a neuromuscular disease. Network are California Pacific Medical
Medical questions should be directed to Center in San Francisco; Columbia
the MDA clinic team, and MDA staff can University in New York; Emory University
provide that contact information if needed. in Atlanta; Massachusetts General in
Boston; and Methodist Hospital in
In most areas, your primary contact at
Houston.
MDA will be the health care service coor-
dinator (HCSC), who can connect you with
all appropriate MDA services and provide
The Clinic Team
information about federal, state and local MDA clinics utilize a multidisciplinary team
resources that may prove helpful. approach, meaning individuals can see
knowledgeable health care specialists from
The MDA Clinic a variety of disciplines, all at one location.
“The MDA clinics have been a very MDA maintains a network of some 200 Specialists can include:
positive part of our lives, and they’ve specialized clinics across the United States n neurologists
really, truly become a part of our fam-
ily. I really can’t imagine what it would
and in Puerto Rico. Most MDA clinics are n cardiologists
be like for our son and for us if there located in teaching hospitals, and many n physiatrists
weren’t an MDA clinic.” MDA clinic directors are university medi-
n orthopedists
Sean Masters, Arizona cal school professors as well as practicing
n psychologists
Parent of a child affected by physicians. MDA clinics are at the fore-
spinal muscular atrophy n pulmonologists
front of research and treatment methods;
n social workers
some clinics also serve as sites for clinical
trials of the latest experimental therapies. n physical and occupational therapists
n nurse case managers
Local MDA staff can direct you to the n speech/language pathologists
closest MDA clinic, or you can find a local n dieticians
MDA clinic online at www.mda.org/locate, n genetic counselors
or by calling (800) 572-1717.
The MDA health care service coordinator
MDA has more than 35 MDA/ALS centers (HCSC) is a central figure at clinic visits.
across the country. MDA distinguishes He or she is usually present on clinic days
some clinics as MDA/ALS centers because to answer questions, distribute MDA edu-
of the medical team’s particular expertise cational materials, coordinate any MDA
with the disease and the research taking services you may require and assist with
place there. community resource referrals.
5 MDA Services Brochure • ©2011 MDA

6. The Diagnostic Process likely undergo an array of testing to ensure
that as much information as possible is
The first step in medical care is determin- obtained and other diseases are ruled out.
ing the nature of the disease. The MDA Many treatment options are dependent on
clinic team can perform diagnostic exami- obtaining a specific diagnosis.
nations and recommend pertinent labora-
tory tests. Among the various types of diagnostic
tools your medical team may use are:
Some of these tests may be extensive, yet
in most instances they can be done on an Clinical examination
outpatient basis. Information about genet- The clinical examination of someone
ic-based diagnostic testing is available at suspected of having a neuromuscular dis-
the MDA clinic. ease focuses on muscle appearance and
strength. Muscles are examined with spe-
Following clinical examination and analysis cial attention given to those of the arms,
of the laboratory tests, many neuromuscu- legs, shoulders and hips. A few neuro-
lar diseases can be quickly and accurately muscular diseases affect facial muscles,
identified. Some neuromuscular diseases, and these too are examined.
however, can be more difficult to diag-
nose. In these cases, the physician will Determining which muscles are not weak
make what is called a “differential diag- is as important as determining which
nosis,” listing two or more diseases that muscles are weak. Each neuromuscular
have similar symptoms. disease typically shows a specific pattern
of muscle involvement. A final diagnosis
A definitive diagnosis may require waiting is based in large part on the pattern of
until the disease has progressed to a stage muscle involvement detected during the
that is unique to that disease. clinical examination.
With the majority of neuromuscular dis- Family history
eases, the first noticeable symptom is
Many neuromuscular diseases are genetic
usually a persistent weakness in one or
in origin. Knowledge of other affected
more muscles.
family members can help to establish and/
“When I was diagnosed with muscular Muscles can become weak for many or confirm a diagnosis. However, it’s pos-
dystrophy, we didn’t know where to sible to have a genetic disease even with-
turn. But the Muscular Dystrophy reasons. The first question the physician
will seek to answer in trying to establish out a family history of that disease.
Association was really there for
me and my family. They walked us a diagnosis is whether muscle function
through what the disease was, what Nerve conduction studies
is abnormal because there is a disease of
we could expect, how we should work and electromyogram
together as a team to tackle this illness muscle itself, or whether muscle function
These two tests often are performed at
and make sure that it didn’t impair me is abnormal because of a disorder that has
from achieving my dreams.” the same evaluation. The nerve conduc-
developed in other tissue (e.g., nerve).
tion studies measure the ability of nerves
Vance Taylor, Maryland The following three pages describe the
Limb-girdle muscular dystrophy to conduct impulses to muscle; this is
most common diagnostic and laboratory
an important test when evaluating for
procedures used to determine why muscle
disorders such as Charcot-Marie-Tooth
function is abnormal and arrive at a defini-
disease. An electromyogram (EMG) mea-
tive diagnosis.
sures electrical activity of muscle. Muscle
Diagnostic Procedures of someone with a neuromuscular disease
Neuromuscular diseases can present in a may have an electrical activity charac-
variety of ways and at different ages. Prior teristically different from that of normal
to receiving a confirmed diagnosis, you’ll muscle.
6 MDA Services Brochure • ©2011 MDA

7. Serum enzyme tests malities in a number of proteins within
Serum enzyme tests measure the amount muscle cells.
of muscle proteins present in the blood.
When muscle tissue is healthy, these Managing Neuromuscular
enzymes, particularly creatine kinase (CK), Disease
remain in muscle and the amount present
in blood is relatively low. Upon diagnosis, a number of services may
be recommended by the MDA clinic team
Many, but not all, neuromuscular diseases as measures to medically manage neuro-
that cause muscle destruction lead to a muscular disorders.
significant increase in the muscle CK and
other enzyme levels found in the blood. Periodic re-evaluations
Thus, serum enzyme tests can be impor- Follow-up visits are provided at MDA clin-
tant aids in the diagnosis of neuromuscu- ics. Ongoing medical management can
lar diseases. include therapies and measures for con-
trolling symptoms, and medical interven-
The value of these tests is often great- tions to assist individuals in maintaining
est at the early stages of the disease. At the highest possible quality of life.
the earliest disease stage, muscle mass
is relatively great and changes in serum Therapy
enzyme levels may occur even before An MDA clinic physician may recommend
symptoms such as weakness become that you consult with a physical, occupa-
apparent. In some neuromuscular dis- tional, respiratory and/or speech therapist
eases, muscle enzyme levels fluctuate with to assist you in managing symptoms of
disease activity. your neuromuscular disease and to main-
tain independence.
At later disease stages, however, muscle
mass may be so reduced that serum Physical therapy: MDA will assist with
enzyme levels may even appear normal. payment for one consultation annually to
(a) evaluate the need for physical therapy
Genetic tests and (b) instruct family members and
A small amount of blood can be used others on how to administer prescribed
to extract DNA from blood cells. This is exercises. Physical therapy can keep still-
extremely valuable for diagnosing genetic healthy muscles functioning, help you
mutations which can cause specific neuro- maintain idependence and prevent the
muscular diseases. onset of painful muscle contractures.
Muscle biopsy Occupational therapy: MDA will assist
In this procedure, muscle tissue is surgi- with payment for one consultation annu-
cally removed for microscopic and/or bio- ally as prescribed. Occupational therapy
chemical analysis. For some neuromus- enables people to make maximum use of
cular diseases, a final diagnosis depends their physical capabilities through the use
on the analysis of a muscle biopsy. The of strategies and techniques, specially
amount of muscle removed is roughly designed implements and daily living aids
equivalent in size to the tip of a little in the home and work environments.
finger. In some conditions, such as the
inflammatory myopathies and central core Respiratory therapy: MDA will assist with
disease, the muscle tissue has a charac- payment for one consultation annually for
teristic appearance under the microscope. instruction in the use of prescribed respi-
Muscle tissue can be analyzed for abnor- ratory therapy equipment designed to aug-
ment or increase vital lung capacity.
7 MDA Services Brochure • ©2011 MDA

8. Speech therapy: When prescribed by an Local MDA offices offer regularly sched-
MDA clinic physician, MDA will assist with uled support group sessions that provide
payment for one consultation annually a chance to meet and share with others
to evaluate the muscles responsible for facing similar challenges.
speech and swallowing. Through such an
evaluation, a speech-language pathologist Support services also are provided
can determine if exercise, use of an aug- online (www.mda.org) through the
mentative communication device and/or lively e-community myMDA and through
modifications to meals are appropriate. myMuscleTeam, which enables families to
keep friends and loved ones updated and
Social services assists in coordinating caregiving tasks.
Social services are a vital aspect of (For more on these programs, see page
the MDA clinic program. MDA local 11.)
offices and clinics may provide additional
resources for families seeking to identify Equipment Assistance
alternate sources of payment for medical
Equipment Program
services and equipment needs.
MDA assists individuals with obtaining
Social workers also may assist with con- medical equipment through its national
necting you to other community resources equipment loan program and referrals to
and providing emotional support for you community resources.
and your family.
MDA’s national equipment program is
Genetic counseling available to anyone affected by the dis-
Genetic counselors can assist families by: eases in MDA’s program for whom medi-
n obtaining and interpreting a genetic cal equipment has been recommended
(DNA) or other appropriate test; through the MDA clinic.
n obtaining a diagnosis for a genetic
To the extent feasible and when available,
condition; MDA provides good-condition refurbished
“There’s a multitude of support groups n determining whether parents of an
that the MDA provides. We’re able to wheelchairs and other durable medical
sit down in a private setting and share affected child are carriers of the equipment medically prescribed due to an
our experiences with other families. By disorder; individual’s neuromuscular disease. This
doing so we realize that we’re not alone
n assessing the risk of passing on the includes wheelchairs, hospital beds, walk-
and we actually realize that if we can
start sharing information with others disease to future children; and ers and canes, communication devices
and helping others, that we can n determining whether other family and similar items.
also help ourselves.”
members may be at risk.
Jeff Vittek, California Through its local field offices, MDA grate-
Parent of a child affected by Flu inoculations fully accepts donations of medical equip-
myotonic muscular dystrophy Influenza is particularly hazardous to ment for distribution through its equip-
people whose respiratory muscles have ment program. MDA is able to make minor
been weakened by neuromuscular disease. repairs to gently used equipment donated
Therefore, MDA assists with flu inocula- to the Association for this purpose.
tions when medically prescribed.
Many federal, state and local resources
exist to assist individuals with equipment
MDA Support Services
purchases. Local MDA offices maintain an
Support services help those with neu- extensive database of resources available
romuscular diseases, their families and in your area. You can contact your local
caregivers address the challenges that MDA office at (800) 572-1717 to be con-
accompany these conditions. nected with knowledgeable staff who will
8 MDA Services • ©2011 MDA

9. help you navigate the process of obtaining the Association annually.
prescribed equipment.
Community resources
Equipment Repairs Services not provided by MDA are often
MDA provides help with the cost of repairs available from other organized community
to all types of medical equipment required agencies. MDA assists in securing help
due to an individual’s neuromuscular from these community resources.
disease. To request such assistance, it’s
important to contact your local MDA office
before repairs are completed. The amount
allowable toward repairs is established by
MDA is Information
M DA is an excellent source of current, n Contact information for all MDA clinics
accurate and practical information and local offices
about all the neuromuscular diseases in its n Tools to help you get involved in the
program, and about daily life with muscle fight against neuromuscular disease
weakness. Information is available in print,
video and online. MDA/ALS Newsmagazine
(alsn.mda.org)
MDA Website
Published bimonthly, this award-winning
(www.mda.org) publication is free to people affected by
This site contains thousands of pages of ALS. In addition to research news and
in-depth information, including: analysis, it includes practical information
n Information about each disease in for individuals and families living with the
MDA’s program, explaining the under- disorder.
lying biology, symptoms, interventions,
inheritance patterns, current research Quest Magazine
and other relevant information (quest.mda.org)
n News updates on research advances
and health care issues This award-winning quarterly publication
is free to those MDA serves. Each issue
n Searchable current and back issues of
“One of the things that we’re really contains articles about research, health
MDA’s magazines (Quest and the MDA/
keeping up on is the status of research. and living with muscle disease, including
That’s a big deal for us and there’s ALS Newsmagazine)
parenting, caregiving, independent living,
always updates on research that come n Printable versions of all MDA publica-
through either Quest magazine helpful products and strategies, legislation
tions
or the MDA website.” and profiles of interesting people.
n State-specific resource guide and links
Bill Stehling, Arizona to nationwide resources
Parent of a child affected by
n Advocacy information about pending
Other MDA Publications
Duchenne muscular dystrophy
legislation relevant to the MDA com- MDA’s comprehensive “Facts About”
munity series provides a concise, informative
n Information about clinical trials and overview of each of the more than 40 dis-
MDA research grants eases in its program. Other publications
n Information about MDA services, pro- include detailed guide books for caregiv-
grams, resources and spokespeople ers, parents and teachers, and booklets
about coping with medical and daily living
9 MDA Services • ©2011 MDA

10. challenges. All publications are free to n Breathe Easy: A Respiratory Guide for
those served by MDA and can be down- People Living with Neuromuscular
loaded online or obtained through your Diseases (video)
local MDA office. n Breath of Life (video for medical pro-
fessionals)
Videos
MDA offers several professionally pro-
duced videos covering medical issues of
interest to people with muscle diseases.
These include:
n With Hope and Courage: Your Guide to
Living with ALS (DVD)
MDA is Empowerment
MDA’s National Task Force on Public
Advocacy Awareness is a group of volunteers who
An important way to get involved with advise the Association on issues of inter-
the MDA community is through the est and importance to people with dis-
Association’s advocacy program. Based abilities. The Task Force consists of adults
in Washington D.C., the advocacy team who are leaders in their communities and
monitors and supports programs, leg- who are affected by one of the neuromus-
islation and health care policies relevant cular diseases in MDA’s program.
to people with neuromuscular diseases,
such as medical care, insurance, acces- Since MDA began, adults with neuro-
sibility, transportation, independent living, muscular diseases have been among its
personal assistance services and research leaders as members of the board of direc-
funding. tors and advisers. Under their guidance,
MDA has educated the public about living
Sign up to be an MDA Advocate on the with disabilities, and supported efforts to
“My ultimate happiness was found in MDA website (www.mda.org/advocacy) provide people with disabilities with equal
the world of advocacy. I encourage and receive e-updates and up-to-the-min- rights and opportunities.
anyone who is thinking about taking on ute news on policy issues impacting the
a leadership role in advocacy. It is very National legislation to which MDA lent
rewarding. Receiving help is an art ... MDA community.
Through advocacy I can give back.” leadership and support include:
MDA’s “Take 5” advocacy initiative
Angela Wrigglesworth, Texas n Muscular Dystrophy Community,
encourages everyone to “take five min-
spinal muscular atrophy
utes” to contact their elected officials Research and Education (MD-CARE)
using online tools that provide talking Amendments Act (passed into law);
points about important legislation and n Spinal Muscular Atrophy Treatment
contact information for elected officials. Acceleration Act;
n Genetic Information Nondiscrimination
MDA’s summer advocacy outreach pro- Act (passed into law);
gram — the MDA Fly Out — provides the n Medicare Improvements to Patients
MDA community with opportunities to and Providers Act (passed into law);
meet with elected officials face to face to
n Access to Complex Rehabilitation and
discuss matters of personal importance.
Assistive Technology Act; and
n ALS Registry Act (passed into law).
10 MDA Services • ©2011 MDA

11. MDA is Community
Summer Camp MDA Chats
MDA camp is a magical place offering a MDA hosts a variety of regularly sched-
wide range of activities specially designed uled online chat sessions at www.mda.
for youngsters ages 6 to 17 who have a org/chat. The chats, which can be disease-
neuromuscular disease. Camp offers an specific, role-specific (such as for parents
unmatched opportunity to develop lifelong or caregivers) or theme-related (such
friendships, share interests and build self- as “positive thinking”), enable real-time
confidence. Activities are geared to the interaction from the comfort of your home
abilities of the campers and range from with other adults who are living with neu-
outdoor sports such as swimming, boat- romuscular diseases.
“MDA camp is basically one of the
most amazing places a child with mus- ing, baseball and horseback riding, to less
cular dystrophy can go. You can do physically demanding programs like arts Volunteer
anything from sports to crafts. We have and crafts, and talent shows. Volunteer
a carnival, a dance. We ride horses. We Volunteers are needed for activities and
have a petting zoo. We have a talent counselors work one on one with camp-
events that raise awareness about neu-
show. I mean, it’s just amazing.” ers around the clock, providing the care,
romuscular diseases and raise funds for
Caroline, MDA camper close supervision and attention the camp-
MDA’s research and services programs.
ers need. Thousands of youngsters attend
Many MDA volunteers find that being part
MDA camp each year at no cost to their
of a larger effort to defeat neuromuscular
families.
diseases fortifies and renews their spirits.
Every local MDA office has a full calendar
myMDA (mda.org/myMDA) of events that depends on volunteers, and
MDA’s online community enables those there also are opportunities for volunteers
affected by neuromuscular diseases to to help with administrative tasks — just
come together and share their experiences ask!
with people who truly understand, while
MDA volunteer opportunities include:
also learning from the experiences of oth-
n Golf tournaments
ers. Members (who must be age 18 or
older) can post photos, videos and blogs, n MDA Muscle Walk
and participate in lively message boards n Hop-A-Thon® (disability awareness/
and disease-specific groups. fundraising program for young chil-
“Through MDA we’ve met so many
people who have been a great support. dren)
The people who work for MDA have
really truly become our friends and part myMuscleTeam n MDA Lock-Up (local business and
community leaders are “arrested” and
of our support network.” (mda.org/mymuscleteam) must raise their “bail”)
Scott and Kirsten Stafne, Minnesota MDA’s myMuscleTeam is a simple, free,
amyotrophic lateral sclerosis n Youth Events (school clubs and organi-
powerful tool that helps MDA families zations combine fun with fundraising)
maximize their most important resource — n MDA Labor Day Telethon local broad-
their “Muscle Team” of family and friends. casts
Users create private, secure myMuscle-
Team Web pages that enable them to To learn more, contact your local office or
post journal entries to keep friends and visit the “How to Help” section of www.
loved ones updated; and utilize a “care mda.org.
coordination calendar” where they can
post requests for assistance and schedule
volunteer help.
11 MDA Services • ©2011 MDA

12. MDA’s Purpose and Programs
T he Muscular Dystrophy Association
fights neuromuscular diseases through
an unparalleled worldwide research effort.
Metabolic Diseases of Muscle
Phosphorylase deficiency (McArdle disease)
Acid maltase deficiency (Pompe disease)
The following diseases are included in Phosphofructokinase deficiency
MDA’s program: (Tarui disease)
Debrancher enzyme deficiency
Muscular Dystrophies
(Cori or Forbes disease)
Myotonic dystrophy (Steinert disease)
Mitochondrial myopathy
Duchenne muscular dystrophy
Carnitine deficiency
Becker muscular dystrophy
Carnitine palmityl transferase deficiency
Limb-girdle muscular dystrophy
Phosphoglycerate kinase deficiency
Facioscapulohumeral muscular dystrophy
Phosphoglycerate mutase deficiency
Congenital muscular dystrophy
Lactate dehydrogenase deficiency
Oculopharyngeal muscular dystrophy
Myoadenylate deaminase deficiency
Distal muscular dystrophy
Emery-Dreifuss muscular dystrophy Myopathies Due to Endocrine
Abnormalities
Motor Neuron Diseases
Hyperthyroid myopathy
Amyotrophic lateral sclerosis (ALS)
Hypothyroid myopathy
Infantile progressive spinal
muscular atrophy Other Myopathies
(Type 1, Werdnig-Hoffmann disease) Myotonia congenita
Intermediate spinal muscular atrophy Paramyotonia congenita
(Type 2) Central core disease
Juvenile spinal muscular atrophy Nemaline myopathy
(Type 3, Kugelberg-Welander disease) Myotubular myopathy
Adult spinal muscular atrophy (Type 4) Periodic paralysis
Spinal-bulbar muscular atrophy
(Kennedy disease)
Inflammatory Myopathies
Polymyositis
Dermatomyositis
Inclusion-body myositis
Diseases of Neuromuscular
Junction
Myasthenia gravis
MDA’s website is constantly
Lambert-Eaton (myasthenic) syndrome
updated with the latest information
Congenital myasthenic syndromes
about the diseases in its program.
Go to www.mda.org. Diseases of Peripheral Nerve
Charcot-Marie-Tooth disease
Jerry Lewis, National Chairman Friedreich’s ataxia
www.mda.org • (800) 572-1717
Dejerine-Sottas disease
©2011, Muscular Dystrophy
Association Inc.
12 MDA Services • ©2011 MDA