
Hope Through Research
At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.
Grants at a Glance - Winter 2013
-
DMD - Matthew Alexander, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Alexander uses a variety of techniques, including the western gel film blot shown here, to identify expression of miR-48...”
-
OPMD - Ayan Banerjee, Ph.D.
Grant Announced: Winter 2013Disease Type: Oculopharyngeal Muscular Dystrophy (OPMD)“Banerjee’s goal is to better understand how the PABN1 protein is regulated, in order to develop a better understanding...”
-
SMA - Gary Bassell, Ph.D.
Grant Announced: Winter 2013Disease Type: Spinal Muscular Atrophy (SMA)““Our central hypothesis is that SMN protein plays a critical role in facilitating regulation of mRNA transport in moto...”
-
BMD, DMD - Linda Baum, M.D., Ph.D.
Grant Announced: Winter 2013Disease Type: Becker Muscular Dystrophy (BMD)““By using these human muscle cells to screen drug libraries to identify compounds that improve cell function, our aim ...”
-
DMD, BMD - Joseph Beavo, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Recent work from Beavo and others strongly suggests that sildenafil ameliorates much of the dysfunction seen in older md...”
-
ALS, CMT - Martha Bhattacharya, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“The peripheral nervous system of the fruit fly Drosophila melanogaster, shown with synaptic vesicle proteins in green an...”
-
CMD, LGMD - Sebahattin Cirak, M.D.
Grant Announced: Winter 2013Disease Type: Congenital Muscular Dystrophy (CMD)“Using the latest sequencing technology, Cirak will study RNA extracted from muscle biopsies of people with CMD or LGMD t...”
-
ALS, IBM - Benoit Coulombe, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Benoit Coulombe (left) and key collaborators in this MDA project, Philippe Cloutier (center) and Denis Faubert (right), ...”
-
DMD, General MD - Radbod Darabi, M.D., Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“The development of induced pluripotent stem cells “opens a new window of opportunity to use patient-specific pluripote...”
-
DMD - Kay Davies, M.A., Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Effects of treatment with a compound that increases utrophin production can be seen in these images. On the left are mic...”
-
SMA - Christine DiDonato, Ph.D.
Grant Announced: Winter 2013Disease Type: Spinal Muscular Atrophy (SMA)“A and B: Side and abdominal views of a mouse walking on a transparent treadmill belt. C: A digital image of paw prints f...”
-
MMD - Matthew Disney, Ph.D.
Grant Announced: Winter 2013Disease Type: Myotonic Dystrophy (DM)“In this X-ray diffraction image, the pattern of dots can be used to extract information about the size, shape and struct...”
-
ALS - Heather Durham, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“In this image of a motor neuron, the bright dots are clumps containing mutant FUS protein.”
-
ALS - Mohamed Farah, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)““The overall goal of this grant is to investigate whether the capacity of motor nerves to regenerate after insult or d...”
-
CMT - Vera Fridman, M.D.
Grant Announced: Winter 2013Disease Type: Charcot-Marie-Tooth Disease (CMT)“HSAN is a rare genetic neuropathy that causes severe numbness, weakness and ulceration of the feet and hands.”
-
DMD - Madhuri Hegde, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)““Our study will help establish a high-throughput, low-cost screening method which can be implemented in newborn screen...”
-
DMD - Eric Hoffman, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Shown is a section of muscle from a child with Duchenne muscular dystrophy, imaged in different ways. The data show a fi...”
-
ALS - Fenghua Hu, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Mutant TDP43 fragments readily form aggregates (green) in mammalian cells, which are occasionally surrounded by the ubiq...”
-
Muscle Physiology - Masahiro Iwamoto, Ph.D., D.D.S.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Iwamoto has discovered molecules that block two different pathologic processes in muscle: formation of bone within the m...”
-
ALS, IBM - Hong Joo Kim, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)““We seek to recapitulate the full spectrum of MSP, to elucidate the molecular mechanism of pathogenesis, and to identi...”
-
DMD - Deok-Ho Kim, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“Kim’s work is focused on developing materials that serve as scaffolds for growing muscle from stem cells in the labora...”
-
FSHD - Michael Kyba, Ph.D.
Grant Announced: Winter 2013Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“Kyba (right) and colleagues Natalie Schennum (left) and Abhijit Dandapat (middle) will use newly developed “gene-editi...”
-
DD – Laing Distal Myopathy - Leslie Leinwand, Ph.D.
Grant Announced: Winter 2013Disease Type: Distal Myopathies“Mutation of the MYH7 gene causes myosin protein to accumulate in the cytoplasm, as shown here (arrow). The structure of ...”
-
CMT - Ronald K. Liem, Ph.D.
Grant Announced: Winter 2013Disease Type: Charcot-Marie-Tooth Disease (CMT)“Spinal cords from unaffected mice (left), and mice with a mutation in the NFL gene (right) are stained with anti-NFL ant...”
-
CMS - Michael Linhoff, Ph.D.
Grant Announced: Winter 2013Disease Type: Congenital Myasthenic Syndromes (CMS)“Linhoff is studying fish (whose tanks are shown) with mutations in a gene that causes congenital myasthenic syndrome.”
-
DMD - Gordon Lynch, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)““There are important and unresolved questions regarding the potential of heat shock proteins for clinical application ...”
-
ALS - Giovanni Manfredi, M.D., Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Cultured motor neurons (in green) and astrocytes (in red) from the spinal cords of ALS transgenic mice. ”
-
SMA - John Manfredi, Ph.D.
Grant Announced: Winter 2013Disease Type: Spinal Muscular Atrophy (SMA)“Manfredi and colleagues have identified several small molecules that promote the growth of axons of motor neurons, which...”
-
MMD - Darren Monckton, Ph.D.
Grant Announced: Winter 2013Disease Type: Myotonic Dystrophy (DM)“The number of CTG repeat units often grows in each successive generation, as shown here, with disease onset occurring ea...”
-
FA - Marek Napierala, Ph.D.
Grant Announced: Winter 2013Disease Type: Friedreich's Ataxia (FA)“Neurons (nerve cells) containing the Friedreich’s ataxia mutation are shown in this microscope image.”
-
ALS - Li Niu, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Finding inhibitors to control the excessive AMPA receptor activity has been a long-pursued strategy for developing AL...”
-
FA - Mark Payne, M.D.
Grant Announced: Winter 2013Disease Type: Friedreich's Ataxia (FA)“Restoring the frataxin protein to FA mice (as shown in the right panel) increases the numbers of mitochondria (M) and sa...”
-
ALS - Sunitha Rangaraju, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Rangaraju will be testing compounds that extend life span in a worm model of ALS, a step in developing drugs that can co...”
-
MG - David Richman, M.D.
Grant Announced: Winter 2013Disease Type: Myasthenia Gravis (MG)“Normal neuromuscular junction (synapse) with the nerve side of the synapse stained green and the muscle side stained red...”
-
ALS, IBM - Eric Ross, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Yeast makes a powerful model system in which to study the spread of prion-like proteins, Ross says, because it grows rap...”
-
ALS - Jeffrey Rothstein, M.D., Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“By comparing cells derived from people with ALS to those from unaffected individuals, Rothstein hopes to find a molecula...”
-
MM – Mitochondrial Myopathies - Eric Schon, Ph.D.
Grant Announced: Winter 2013Disease Type: Mitochondrial Myopathies (MM)“Schon is seeking ways to induce cells to selectively degrade their mutant mitochondria as a potential treatment of mitoc...”
-
FSHD - Rabi Tawil, M.D.
Grant Announced: Winter 2013Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“Tawil will be searching for biological markers for FSHD that can be used in clinical trials of future therapeutics. “R...”
-
DMD - Joan Taylor, Ph.D.
Grant Announced: Winter 2013Disease Type: Duchenne Muscular Dystrophy (DMD)“GRAF1 promotes muscle formation in a dish. Immature muscle cells fuse to form multinucleated muscle fibers during format...”
-
MG - Muthusamy Thiruppathi, Ph.D.
Grant Announced: Winter 2013Disease Type: Myasthenia Gravis (MG)“It may be possible to “change the approach to the treatment of myasthenia gravis from global, nonspecific immune modul...”
-
MG - Socrates Tzartos, Ph.D.
Grant Announced: Winter 2013Disease Type: Myasthenia Gravis (MG)“Tzartos will develop a clinically practical tool for diagnosis of myasthenia gravis that is caused by the development of...”
-
ALS - Xin Wang, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Wang will explore the potential therapeutic value of melatonin-like drugs in slowing the progression of ALS.”
-
LGMD - Noah Weisleder, Ph.D.
Grant Announced: Winter 2013Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“Noah Weisleder (left) in his laboratory working with Eric Beck (center) and Jenna Alloush (right) on a fluorescence micr...”
-
OPMD - Sarah Youssof, M.D.
Grant Announced: Winter 2013Disease Type: Oculopharyngeal Muscular Dystrophy (OPMD)“Youssof is learning about patients’ perspectives on OPMD and studying outcome measures for incorporation into clinical...”
-
ALS - Daniela Zarnescu, Ph.D.
Grant Announced: Winter 2013Disease Type: Amyotrophic Lateral Sclerosis (ALS)“A fruit fly neuromuscular junction showing synaptic terminals (purple) and mutant TDP43 protein forming puncta, or point...”