ViroPharma is set to initiate VP20629 Phase II trial in the second half of 2013

ViroPharma is set to initiate VP20629 Phase II trial in the second half of 2013

ViroPharma: Strengthening Financial Profile & Pipeline Indicate Long-Term Upside, Ivan Deryugin, December 27, 2012

VP20629 is licensed from Intellect Neurosciences for the treatment of Friedreich’s Ataxia (FA), a rare neurodegenerative disease that affects about 1 in 50,000 people. Symptoms of FA include heart disease, speech problems, and gait disturbance. It is estimated that there are 6,000 FA patients here in the United States. Phase I data for VP20629 showed that the drug was well-tolerated at all dose levels, and ViroPharma is set to initiate a Phase II trial in the second half of 2013, and the company plans to file for orphan drug status once Phase II data is available

About VP 20629

Missense Mutations Linked to Friedreich Ataxia Have Different but Synergistic Effects on Mitochondrial Frataxin Isoforms

Missense Mutations Linked to Friedreich Ataxia Have Different but Synergistic Effects on Mitochondrial Frataxin Isoforms . Hongqiao Li, Oleksandr Gakh, Douglas Y. Smith IV, Wasantha K Ranatunga and Grazia Isaya. J. Biol. Chem. jbc.M112.435263. First Published on December 26, 2012, doi:10.1074/jbc.M112.435263

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Mammalian frataxin controls sulfur production and iron entry during de novo Fe4S4 cluster assembly

Mammalian frataxin controls sulfur production and iron entry during de novo Fe4S4 cluster assembly. Florent Colin , Alain Martelli , Martin Clémancey , Jean-Marc Latour , Serge Gambarelli , Laura Zeppieri , Catherine Birck , Adeline Page , Hélène Puccio , and Sandrine Ollagnier de Choudens. J. Am. Chem. Soc., Just Accepted Manuscript, DOI: 10.1021/ja308736e. Publication Date (Web): December 22, 2012

Keywords: Fe-S clusters, cysteine desulfurase complex (NFS1/ISD11), frataxin (FXN), Freidreich’s ataxia, cysteine desulfurization, aconitase (mACO2).

Diabetes Mellitus in Children and Adolescents with Genetic Syndromes.

Diabetes Mellitus in Children and Adolescents with Genetic Syndromes. Schmidt F, Kapellen TM, Wiegand S, Herbst A, Wolf J, Fröhlich-Reiterer EE, Rabl W, Rohrer T, Holl RW; Experimental and Clinical Endocrinology & Diabetes : Official Journal, German Society of Endocrinology [and] German Diabetes Association [2012, 120(10):579-585]

Keywords: genetic syndromes, diabetes mellitus (DM), frequency, treatment strategies, long-term complications, Turner syndrome (TS), Prader-Willi syndrome (PWS), Friedreich ataxia (FA), Alström syndrome (AS), Klinefelter syndrome (KS), Bardet-Biedl syndrome (BBS), Berardinelli-Seip syndrome (BSS), Down syndrome (DS).

USF researchers study medication that may help patients with incurable neuro-muscular disorder

USF researchers study medication that may help patients with incurable neuro-muscular disorder. abc ACTION NEWS; Thursday, December 20, 2012

“Currently there are no treatments or cures for Friedreich's Ataxia, but there's a possibility for a new drug that USF will be spearheading in the next month called EPI – 743"

Kinetics of the Triplex-Duplex Transition in DNA

Kinetics of the Triplex-Duplex Transition in DNA. Il-Buem Lee, Seok-Cheol Hong, Nam-Kyung Lee, Albert Johner; Biophysical Journal, Volume 103, Issue 12, 19 December 2012, Pages 2492-2501. http://dx.doi.org/10.1016/j.bpj.2012.10.029

Keywords: triplex folding/unfolding, metastable states.

Clarification: Conditions like FA involve triplex DNA structures that arise from tri nucleotide repeat expansions.

Heart Mitochondria: Receivers and Transmitters of Signals

Heart Mitochondria: Receivers and Transmitters of Signals. José Marín-García M.D.;
Mitochondria and Their Role in Cardiovascular Disease
2013, pp 157-181

Keywords: ATP, heart mitochondria, regulatory and signaling events, heart failure (HF), myocardial ischemia and reperfusion injury (IRI), hypoxia, oxidative stress (OS), hormonal and cytokine stimuli, interorganelle communication, hypertrophic growth, cell death, interrelated signaling pathways.

Alkylaminoquinones as Multifunctional Radical Quenchers

Alkylaminoquinones as Multifunctional Radical Quenchers. The Biodesign Institute Arizona State University.

Researchers at the Biodesign Institute of Arizona State University have developed novel
multifunctional radical quenchers (MRQs) for the treatment of mitochondrial disorders
Therapeutic candidates for mitochondrial diseases such as Friedreich’s ataxia

Extramembranous and Interosseous Technique of Tibialis Posterior Tendon Transfer

Extramembranous and Interosseous Technique of Tibialis Posterior Tendon Transfer.ClinicalTrials.gov Identifier: NCT01751503; First received: December 11, 2012

Iva Hauptmannova, Royal National Orthopaedic Hospital NHS Trust, London, Middlesex, United Kingdom

Foot drop deformity is a life limiting condition characterized by loss of ankle dorsiflexion and eversion. Main condition leading to drop foot condition include irrecoverable muscle and nerve injuries, poliomyelitis, drug poisoning, strokes, cerebral palsy, Charcot - Marie - Tooth disease, meningomyelocele, club foot, Friedreich's ataxia and Leprosy.

High-performance neuroprosthetic control by an individual with tetraplegia

High-performance neuroprosthetic control by an individual with tetraplegia. Jennifer L Collinger PhD, Brian Wodlinger PhD, John E Downey BS, Wei Wang PhD, Elizabeth C Tyler-Kabara MD, Douglas J Weber PhD, Angus JC McMorland PhD, Meel Velliste PhD, Prof Michael L Boninger MD, Prof Andrew B Schwartz PhD. The Lancet, Early Online Publication, 17 December 2012, doi:10.1016/S0140-6736(12)61816-9


Paralyzed Woman's Mind Controls Robotic Arm. Written by Sarah Glynn, Medical News Today, Article Date: 17 Dec 2012.

A paralyzed woman struggling with Spinocerebellar degeneration has been able to control a robotic prosthetic hand using her thoughts.