Neuromuscular Diseases : Focus on Multiple Sclerosis , Amyotrophic Lateral Sclerosis , and Muscular Dystrophy. 9.20 - 9.30 Introduction to the session
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9.20 - 9.30Introductionto the session
9.30 - 10.15 Alex KAZANTSEV – Massachusetts General Hospital, Harvard MedicalSchool, Charlestown, MA (USA) Multiple and AmyotrophicLateralSclerosis : Understanding the MechanismsofNeurodegeneration and Developing Diverse Therapeutics
10.15 - 11.00 John MATSOUKAS - UniversityofPatras, Patras (Gr)Linear and CyclicMyelinAnalogues in the Immunotherapyof Multiple Sclerosis
11.30 - 12.15 Rebecca PRUSS - TROPHOS, Marseille (F)UseofCellBasedAssaystoDefine SAR and SelectDrugCandidatesfor Motor NeuronDiseases (and OtherIndications)
15.00 - 15.45 Pier Lorenzo PURI - BurnhamInstitute, San Diego (USA) e Dulbecco Telethon Institute, S. Lucia/EBRI , Rome (I)EpigeneticBasisofMuscleRegeneration in MuscularDystrophy
15.45 - 16.30 Carlo GAETANO - IDI, Rome (I) NO Sparks off Chromatin: Tale of a MultifacetedEpigeneticRegulator
Multiple sclerosis (or MS) is a chronic, often disabling disease
That attacks the central nervous system (CNS), which is made up of
the brain, spinal cord, and optic nerves. Symptoms may be mild,
such as numbness in the limbs, or severe, such as paralysis or loss
The progress, severity, and specific symptoms of MS are
unpredictable and vary from one person to another. Today, new
treatments and advances in research are giving new hope to people
affected by the disease.
The body’s own defense system attacks myelin, the fatty substance
that surrounds and protects the nerve fibers in the central nervous
system. The nerve fibers themselves can also be damaged.
The damaged myelin forms scar tissue (sclerosis), which gives the
disease its name. When any part of the myelin sheath or nerve fiber
is damaged or destroyed, nerve impulses traveling to and from the
brain and spinal cord are distorted or interrupted, producing the
variety of symptoms that can occur.
Although there is still no cure for MS, effective strategies are available
to modify the disease course, treat exacerbations (also called attacks,
relapses, or flare-ups), manage symptoms, improve function and
safety, and provide emotional support. In combination, these
treatments enhance the quality of life for people living with MS.
Glatiramer acetate is a random polymer (average molecular mass 6.4 kD) composed of four amino acids that are found in myelin basic protein. It is an immunomodulator, licensed in much of the world for reduced frequency of relapses in relapsing-remitting multiple sclerosis.
It is a non-interferon and non steroidal medication. The mechanism of action for glatiramer is unknown, although several have been proposed.
Mitoxantrone is a type II topoisomeraseinhibitor; it disrupts DNA synthesis and DNA repair in both healthy cells and cancer cells.
It also engages in intercalation.
It is used in the treatment of certain types of cancer, mostly metastaticbreast cancer, acute myeloid leukemia, and non-Hodgkin's lymphoma.
Mitoxantrone is also used to treat multiple sclerosis (MS), most notably the subset known as secondary progressive MS. Mitoxantrone will not cure multiple sclerosis, but is effective in slowing the progression of secondary progressive MS.
Amyotrophic lateral sclerosis (ALS) is a progressive
neurodegenerative disease that affects nerve cells in the brain
and the spinal cord.
Motor neurons reach from the brain to the spinal cord and from
the spinal cord to the muscles throughout the body.
The progressive degeneration of the motor neurons in ALS
eventually leads to their death. When the motor neurons die,
the ability of the brain to initiate and control muscle movement is lost.
With voluntary muscle action progressively affected, patients in the
later stages of the disease may become totally paralyzed.
No cure has yet been found for ALS. However, the FDA has
approved the first drug treatment for the disease—riluzole.
Riluzole is believed to reduce damage to motor neurons and prolongs
survival by several months, mainly in those with difficulty swallowing.
Other treatments are designed to relieve symptoms and
improve the quality of life for people with ALS. Drugs also are available to help individuals with pain, depression, sleep disturbances, and constipation. Individuals with ALS may eventually consider forms of mechanical ventilation (respirators).
Muscular dystrophy (MD) is a genetic disorder that weakens the
muscles that help the body move. People with MD have incorrect
or missing information in their genes, which prevents them from
making the proteins they need for healthy muscles. MD weakens
muscles over time, so children, teens, and adults who have the
disease can gradually lose the ability to do the things most people
take for granted, like walking or sitting up.
Someone with MD might start having muscle problems as a baby
or their symptoms might start later.
Some people even develop MD as adults.
Duchennemuscular dystrophy(DMD), the most common type
of the disease, is caused by a problem with the gene that makes a
protein called dystrophin. This protein helps muscle cells keep their
shape and strength. Without it, muscles break down and a person
gradually becomes weaker.
There's currently no cure for any form of muscular dystrophy.
Research into gene therapy may eventually provide treatment to stop the progression of some types of muscular dystrophy.
Current treatment is designed to help prevent or reduce deformities
in the joints and the spine and to allow people with MD to remain
mobile as long as possible. Treatments may include various types of
physical therapy, medications, assistive devices and surgery.
MedicationsDoctors prescribe medications to treat some forms of MD:
Myotonic dystrophy. Medications that may be used to help manage
the muscle spasms, stiffness and weakness associated with this
condition include mexiletine, phenytoin, baclofen, dantrolene
Duchenne's muscular dystrophy. The anti-inflammatory corticosteroid
medication prednisone may help improve muscle strength and delay
the progression of Duchenne's MD.