Answers to NL quiz
NL #30 - September 2010
1. In a recent article published in Science, Edgar Gomes and colleagues have recently identified a new structure in the nucleus of the cell. What is this structure called?
a) TEN
b) NET
=> c) TAN
d) NAT
a) TEN
b) NET
=> c) TAN
d) NAT
2. Committed fibroblasts have recently been reprogrammed into which cell type?
a) adipocytes
b) astrocytes
c) myoblasts
=> d) cardiomyocytes
3. The Phase III clinical trial conducted by Genzyme assessed the efficacy of Myozyme for the treatment of which disease?
=> a) Pompe disease
b) Duchenne Muscular Dystrophy
c) Spinal Muscular Atrophy
d) Limb-Girdle Muscular Dystrophy
4) Researchers at the Stanford University School of Medicine have developed a novel method to grow adult stem cells in culture using
a) xerogel
=> b) hydrogel
c) organogel
d) aerogel
5) How many different treatment regimens for Duchenne muscular dystrophy are used by doctors around the world?
=> a) 29
b) 24
c) 27
d) 28
NL #29 - June 2010
1. In the ULENAP clinical trial, which tool was specially designed at the Institute of Myology for non-ambulatory neuromuscular patients?
a) Taping
=> b) Moviplate cylinders
c) Handgrip
d) Myowrist
a) Taping
=> b) Moviplate cylinders
c) Handgrip
d) Myowrist
2. Several research groups have allied with industry partners to launch a large European collaborative project called
=> a) EndoStem
b) MyoStem
c) EndoCell
d) MyoCell
=> a) EndoStem
b) MyoStem
c) EndoCell
d) MyoCell
3. In the study of the experimental drug, GSK2402968 under development by GlaxoSmithKline, which route of administration was employed?
a) intramuscular
b) intravenous
=> c) subcutaneous
d) intracardiac
a) intramuscular
b) intravenous
=> c) subcutaneous
d) intracardiac
4. Which GTPase enzyme found in the outer mitochondrial membrane is involved in Charcot Marie tooth type 2A?
=> a) mitofusin 2
b) Ras p21
c) Gyp1p
d) Rab6
=> a) mitofusin 2
b) Ras p21
c) Gyp1p
d) Rab6
5. Which chemical has recently been found to reduce the symptoms of OPMD in a mouse model of the disease?
a) cysteine
b) cytosine
c) citrulline
=> d) cystamine
a) cysteine
b) cytosine
c) citrulline
=> d) cystamine
NL #28 - May 2010
1. In France, treatment of Lambert Eaton syndrome (LEMS) was based on which drug?
a) 3,4-DAP
=> b) guanidine
c) arginine
d) DDP- 4
a) 3,4-DAP
=> b) guanidine
c) arginine
d) DDP- 4
2. Approximately how many individuals in the European Union can be assumed to be currently suffering from LEMS?
=> a) 4,000
b) 10000
c) 6000
d) 3000
=> a) 4,000
b) 10000
c) 6000
d) 3000
3. Which pharmaceutical company has just commenced a phase 2 clinical trial in DMD patients?
a) PTC Therapeutics
=> b) Acceleron Pharma
c) Prosensa
d) BioMarin
a) PTC Therapeutics
=> b) Acceleron Pharma
c) Prosensa
d) BioMarin
4. An X-linked form of distal hereditary motor neuropathies was recently found to be due to mutations in which copper transporter gene?
=> a) ATP7A
b) hCTR1
c) CCS
d) hCOX17
=> a) ATP7A
b) hCTR1
c) CCS
d) hCOX17
5. The therapeutic effect of Glatiramer acetate (GA) was investigated in the dy2J/dy2J mouse model of merosin deficient congenital muscular dystrophy. For which disorder has this drug been approved?
a) Alzheimer’s disease
b) Chronic Fatigue Syndrome
c) Myasthenia gravis
=> d) relapsing-remitting multiple sclerosis
a) Alzheimer’s disease
b) Chronic Fatigue Syndrome
c) Myasthenia gravis
=> d) relapsing-remitting multiple sclerosis
NL #27 - April 2010
1. What type of analysis did the authors use in a recent paper to gain insight into molecular mechanisms involved in OPMD?
a) proteomic analysis
b) genomic analysis
c) PCR analysis
=> d) transcriptomic analysis
a) proteomic analysis
b) genomic analysis
c) PCR analysis
=> d) transcriptomic analysis
2. VLA-4 and/or VLA-5 interact with which of the following extracellular matrix proteins, possibly contributing to T-cell mediated tissue damage?
a) undulin
=> b) fibronectin
c) tenascin
d) laminin
a) undulin
=> b) fibronectin
c) tenascin
d) laminin
3. Intramuscular injections of a dual-adeno-associated vector into a dysferlin-deficient mouse model led to a widespread expression of the full-length protein.
True
=> False
True
=> False
4) In a recent Nature Biotechnology publication, the spinal muscular atrophy phenotype in a mouse model of SMA was rescued using gene therapy. How many days after injection was AAV9 observed in the spinal cord motor neurons?
a) 5 days
b) 21 days
c) 15 days
=> d) 10 days
a) 5 days
b) 21 days
c) 15 days
=> d) 10 days
5) The drafting of the family guidelines ‘The Diagnosis and management of Duchenne muscular dystrophy’ was led by:
=> a) the Centers for Disease Control (CDC)
b) The Muscular Dystrophy Association
c) the United Parent Project Muscular Dystrophy
d) TREAT-NMD
=> a) the Centers for Disease Control (CDC)
b) The Muscular Dystrophy Association
c) the United Parent Project Muscular Dystrophy
d) TREAT-NMD
NL #26 - March 2010
1. A previously unknown type of muscle stem cell has recently been identified. Where is it located?
a) in the sarcolemma
b) in the connective tissue
=> c) in the interstitial spaces of muscle fibres
d) in intramuscular fat
a) in the sarcolemma
b) in the connective tissue
=> c) in the interstitial spaces of muscle fibres
d) in intramuscular fat
2. Why are in vivo evaluations of neuromuscular function a powerful tool?
a) allows to follow the same animal during treatment
b) non-invasive
c) increases the methodological robustness
=> d) all of the above
a) allows to follow the same animal during treatment
b) non-invasive
c) increases the methodological robustness
=> d) all of the above
3. Specific mutations in which gene have been found to cause both Limb Girdle Muscular Dystrophy type 2L (LGMD2L) and Miyoshi Myopathy (MMD3)?
=> a) anoctamin 5
b) dysferlin
c) calpain-3
d) fukutin
=> a) anoctamin 5
b) dysferlin
c) calpain-3
d) fukutin
4. Généthon has initiated a clinical trail for which rare disease?
a) Wissler-Fanconi syndrome
=> b) Wiskott Aldrich syndrome
c) Aldred syndrome
d) Immune-mediated rippling muscle disease
a) Wissler-Fanconi syndrome
=> b) Wiskott Aldrich syndrome
c) Aldred syndrome
d) Immune-mediated rippling muscle disease
5. When AAVs penetrate cells, their genomes tend to combine with one another in a phenomenon known as:
=> a) concatemerisation
b) amalgamation
c) dimerization
d) coalescence
=> a) concatemerisation
b) amalgamation
c) dimerization
d) coalescence
NL #25 - February 2010
1. The U7 pre-clinical project involves the loco-regional injection of the vector-drug ‘AAV-U7’ into the muscle of which animal model:
a) monkey
=> b) dog
c) cat
d) hamster
a) monkey
=> b) dog
c) cat
d) hamster
2. Cardiac manifestations occur in what percentage of individuals with Type 1 Myotonic Dystrophy?
=> a) > 80%
b) 60%
c) <30%
d) 50%
=> a) > 80%
b) 60%
c) <30%
d) 50%
3. A new form of autosomal dominant limb-girdle muscular dystrophy has recently been described in which country?
a) Spain
b) Brazil
c) Italy
d) Tunisia
a) Spain
b) Brazil
c) Italy
d) Tunisia
4) Thyrotoxic hypokalemic periodic paralysis (TPP) is seen predominantly in which of the following populations?
a) Caucasians
b) Hispanics
c) African-Americans
=> d) Asians
a) Caucasians
b) Hispanics
c) African-Americans
=> d) Asians
5) The positive preliminary results from systemic treatment with AVI-4658 were observed after how many weeks of treatment?
=> a) 12 weeks
b) 6 weeks
c) 9 weeks
d) 20 weeks
=> a) 12 weeks
b) 6 weeks
c) 9 weeks
d) 20 weeks
NL #24 - December 2009 / January 2010
1. All of the following are CAG-repeat disorders EXCEPT:
a) Huntington disease
=> b) Myotonic dystrophy (CTG repeat)
c) Olivopontocerebellar atrophy (spinocerebellar ataxia type 2)
d) Kennedy disease (X-linked spinal and bulbar muscular atrophy)
2. Pseudohypertrophy is characterized by:
a) An initial increase in number of muscle cells to compensate for the degenerating muscle cells.
b) Local interstitial oedema.
c) An increase in muscle bulk to compensate for proximal muscle weakness.
=> d) An initial increase in the size of muscle fibres, followed by an increase in muscle volume due to deposits of fat and connective tissue.
(Muscle biopsy of DMD patients demonstrates degeneration and regeneration of muscle fibres, isolated hypertrophic fibres, and significant replacement of muscle by fat and connective tissue. Muscle fibre necrosis and subsequent fibrous changes are intertwined with the inflammatory response, which also causes the initial enlargement of individual muscle fibres. However, the calves appear large due to an increase in deposits of fat and collagen, not due to the inflammatory response or an increasing number of muscle fibres).
3. Amyotrophic lateral sclerosis (ALS) is a disease that causes degeneration of the motor neurons of the central nervous system, resulting in loss of voluntary muscle control, and ultimately death. What is the colloquial name for this disease in the United States?
a) Stephen Hawking's disease
b) David Niven's disease
=> c) Lou Gehrig's disease
d) Charlie Mingus' disease
4. Treatment for muscular dystrophy includes all of the following except:
a) Surgery
b) Physical therapy
c) Corticosteroids
=> d) Botulinum toxin injections
(Treatment for muscular dystrophy seeks to keep the patient independent and mobile for as long as possible and slow the progression of the disease. Treatment can also help prevent or reduce the occurrence of painful deformities and life–threatening complications. Medications, such as corticosteroids, can help delay degeneration of muscles. Medications to control muscle spasms or vitamin D supplements to build strong bones may be taken. Other treatment options may include physical and speech therapy, orthopaedic braces and muscle surgery).
5. Which pharmaceutical company recently received a grant from the AFM for its Omigapil program in Congenital Muscular Dystrophy ?
a) AVI BioPharma
=> b) Santhera Pharmaceuticals
c) Sanofi Aventis
d) GlaxoSmithKline (GSK)
Picture: http://www.mathpuzzle.com/loyd/cop294-295.html
a) Huntington disease
=> b) Myotonic dystrophy (CTG repeat)
c) Olivopontocerebellar atrophy (spinocerebellar ataxia type 2)
d) Kennedy disease (X-linked spinal and bulbar muscular atrophy)
2. Pseudohypertrophy is characterized by:
a) An initial increase in number of muscle cells to compensate for the degenerating muscle cells.
b) Local interstitial oedema.
c) An increase in muscle bulk to compensate for proximal muscle weakness.
=> d) An initial increase in the size of muscle fibres, followed by an increase in muscle volume due to deposits of fat and connective tissue.
(Muscle biopsy of DMD patients demonstrates degeneration and regeneration of muscle fibres, isolated hypertrophic fibres, and significant replacement of muscle by fat and connective tissue. Muscle fibre necrosis and subsequent fibrous changes are intertwined with the inflammatory response, which also causes the initial enlargement of individual muscle fibres. However, the calves appear large due to an increase in deposits of fat and collagen, not due to the inflammatory response or an increasing number of muscle fibres).
3. Amyotrophic lateral sclerosis (ALS) is a disease that causes degeneration of the motor neurons of the central nervous system, resulting in loss of voluntary muscle control, and ultimately death. What is the colloquial name for this disease in the United States?
a) Stephen Hawking's disease
b) David Niven's disease
=> c) Lou Gehrig's disease
d) Charlie Mingus' disease
4. Treatment for muscular dystrophy includes all of the following except:
a) Surgery
b) Physical therapy
c) Corticosteroids
=> d) Botulinum toxin injections
(Treatment for muscular dystrophy seeks to keep the patient independent and mobile for as long as possible and slow the progression of the disease. Treatment can also help prevent or reduce the occurrence of painful deformities and life–threatening complications. Medications, such as corticosteroids, can help delay degeneration of muscles. Medications to control muscle spasms or vitamin D supplements to build strong bones may be taken. Other treatment options may include physical and speech therapy, orthopaedic braces and muscle surgery).
5. Which pharmaceutical company recently received a grant from the AFM for its Omigapil program in Congenital Muscular Dystrophy ?
a) AVI BioPharma
=> b) Santhera Pharmaceuticals
c) Sanofi Aventis
d) GlaxoSmithKline (GSK)
Picture: http://www.mathpuzzle.com/loyd/cop294-295.html
