2nd dart international ( virtual ) conference -2020

Supported by

MONDAY, 7th SEPT 2020 « On Zoom « 10am - 1pm (Indian Standard Time)

Dystrophy Annihilation Research Trust

2nd DART INTERNATIONAL ( VIRTUAL ) CONFERENCE -2020 On World Duchenne Awareness Day (WDAD20)

DART - MISSION CURE - DMD Research ¯ Therapies

https://dartindia.in/wdad20/ FREE REGISTRATION FREE REGISTRATION

www.dartindia.in [email protected] RSVP: 9008516322



ABOUTMUSCULARDYSTROPHYThe Muscular Dystrophies are a group of genetic diseases characterized byprogressiveweaknessanddegenerationoftheskeletalorvoluntarymuscleswhichcontrol movement. The most prevalent and fatal form is Duchenne MuscularDystrophy which is caused due to a lack of a protein called ‘Dystrophin”. TheDiseaseaffectsonlyboysbutgirlsarecarriers.Oneoutof3500boyisaffectedandtherearearound4,50,000childrensufferingfromthisterribledisease.

ABOUTDARTDystrophyAnnihilationResearchTrust(DART)isaparent-poweredNGOfoundedinV2012,withamissiontocreateacommonplatformtoprovidecounsellingandsupportforallMuscularDystrophypatientsandtheirfamilies,aswellastocreateawarenessofTreatmentsandDrugTrials,andlastbutnotleast,facilitateresearchintoalleviatingthescourgeofMusculardystrophy.DARTisuniqueasaparentrunMinistryofScienceandTechnologyGovtofIndiaDSIRcertifiedresearchlab.

ABOUTTHECONFERENCETheconferencewillcatertoAcademicians,Patients,Caregivers,PatientAdvocacyOrganisations,CliniciansandIndustryProfessionalswhowillhavetheplatformto:•NetworkwithpartnersonkeyissuesandchallengesinDMD• Engage with panels of international experts who will inform, challenge andstimulatedebate•Acquirethelatestupdatesonstandardsofcare,outcomemeasures,patientregistriesandnewdevelopments



WelcomeDearFriends,Welcome to our 2ND International(Virtual )Conference on ”World DuchenneAwarenessDay2020".From humble beginnings as an NGO cumResearchorganisationDARThascomea longway. Having conducted many events andactivities, it is indeedaproudmoment forustobeorganisingthisconference. All of you are a source of hope for thepatients and their families. With yourcollective efforts better management andtreatment of Muscular Dystrophy hasenhancedthequalityoflivesofourchildren.Weappreciateyoursupportinunderstandingclinical research formedical innovation and Iamsurevaluable ideaswill emerge from thisconferencethatwillbeofgreatbenefittooneandall.

Dr. Soumya Swaminathan

Professor Steve Wilton

Dr. Arun Shastry

Prof. Vijay Chandru

Dr. Meenakshi Bhat


Dr. Soumya Swaminathan is the current Chief Scientist at the World Health Organization (WHO) since March 2019. She is an Indian pediatrician and clinical scientist known for her research on tuberculosis. Previously she was the Deputy Director General of Programmes (DDP) at WHO and Director General of the Indian Council of Medical Research (ICMR).

Prof Wilton is the Director for the Centre for Molecular Medicine and Therapeutics, a joint venture between the Perron Institute and Murdoch University, Western Australia. With his colleague Prof. Sue Fletcher, he pioneered the development of Exon Skipping, a radical new therapy for Duchenne muscular dystrophy (DMD). He has extended the therapeutic applications of antisense oligonucleotides (AONs) to other inherited and acquired conditions as well.

Prof. Vijay Chandru is an iconic figure in the Bangalore Biotech circle. A UCLA graduate in Engineering Systems and a PhD from the MIT Operations Research Center, he is the founder of the Association of Biotech Led Enterprises (ABLE). He is also an entrepreneur who founded Strand Life Sciences. Prof. Chandru is a fellow of both the Indian Academy of Sciences and the Indian National Academy of Engineers. He has won numerous awards and published many game-changing articles and books.

Dr. Meenakshi Bhat is the head consultant in Clinical Genetics at the Center of Human Genetics (CHG), Bangalore. Her training in the UK included MRCP (Paed.) from Ireland and 4 year super-specialty training in Clinical Genetics from London. CHG offers comprehensive genetic evaluation of families with rare disorders, genetic counselling and diagnostic testing. She also plays a leading role in a first-of-its-kind Rare Diseases Centre of Excellence.

Dr. Arun Shastry has more than 15 years of drug discovery and development experience. A PhD in Immunogenetics from Madras Diabetes Research Foundation (University of Madras), he worked as a Guest Researcher at Karolinska Institute, Sweden. Currently he is the Chief Scientific Officer at DART, spearheading the research and development of AON based Exon Skipping. He is also the Co-Founder of Hanugen Therapeutics focusing on synthesis of clinical Grade Oligonucleotides. He is an expert on the DBT "Mission Program on Management and Treatment of Rare and Genetic Diseases" and a member of TREAT-NMD Alliance Neuromuscular Network.

From the Research World

Dr. J. Andoni Urtizberea


Dr. Andoni is the Scientific Director of the European Neuromuscular Center (ENMC) and former Medical Director of the AFM-Telethon (the French MDA). He played an instrumental role in the establishment of many worldwide networks in myology, an emerging discipline dedicated to muscle and related disorders. He is also a part-time clinical myologist in Hendaye, (Hôpital Marin, APHP), deputy coordinator of the French Neuromuscular Network (FILNEMUS) in Marseilles and, member of the Executive Board of the TREAT-NMD Alliance. Dr. Ishwar C Verma Dr. IC Verma is the senior Consultant and Head, Department of Medical Genetics at Sir Ganga Ram Hospital, New Delhi. He is a Fellow of the Royal College of Physicians, London, the American Academy of Pediatrics, and the National Academy of Medical Sciences. He is a Member and Vice-chairman of the Ethics Committee of the International Human Genome Organization (HUGO) and serves as an adviser in genetics to the WHO.

Dr. Ann Agnes Mathew Dr. Ann is a renowned Consultant Paediatric Neurologist who has worked at the Great Ormond Street Hospital in London and currently consults at Sagar Hospitals, Bangalore and is a visiting consultant for Bangalore Baptist Hospital and St. Martha’s Hospital. She was part of the team who established the first paediatric Neuromuscular service in the country. She runs a regular neuromuscular clinic in Mediscan Chennai for children with DMD, SMA, myopathies, myasthenias and those on the Pompe services.

Dr. Renu Suthar Dr. Renu Suthar is Associate Professor of Paediatric Neurology, Postgraduate Institute of Medical Education and Research (PGIMER), Chandigarh. She specializes in pediatric neuromuscular disorders such as DMD, SMA, Neuropathy and CMDs. She is the Principal Investigator in the Phase 3 Randomized control trial of Ataluren in boys with DMD and Co-PI in safety and efficacy of Deflazacort dose escalation in boys with DMD.

Ms. Jesse Jeffrey Ms. Jesse is a Developmental Therapist at a leading early intervention specialist centre in Bengaluru, India. She specialises in helping children cope with Autism Spectrum Disorder, Social (Pragmatic) Communication Disorder, Specific Learning Disabilities, Cornelia de Lange Syndrome and behavioral challenges.

From the Clinical World


From The Education and Mentor world Dr. Rema Nandakumar

In a career spanning 29 years, Ms Rema Nandakumar has worked as a teacher for 14 years, as the Principal of two prominent schools for 13 years and the Dean of Samsidh Group of Schools for 2 years. According to her, an education should prepare a child for the journey of life that lies ahead. With the firm belief and a clear action plan to make schools an avenue for the holistic development of the child, she has always endeavoured to provide children with the environment and exposure to top-class academic training as well as training in values and life skills, two non- negotiable aspects of a child's education.

Ms. Manju Balasubramanyam Ms Manju is the Principal at Delhi Public School Bangalore North since 2009. She is a keen lifelong learner, technology enthusiast and above all a passionate teacher and empath. She is a die-hard optimist and a firm believer in Inclusion, who believes that diversity enriches the world. She has been bestowed with many awards including the 'National Award to Teachers' in 2017 by Department of Education and Literacy, Ministry of Human Resource Department, Government of India and has been awarded as the ‘Progressive Principal of India’ by the Centre of Educational Development in 2018.

Dr. A Nagarathna Dr. A. Nagarathna is the Principal of M.S Ramaiah College of Arts, Science and Commerce(MSRCASC) and has a flair for updating her knowledge and helps others to do the same. She has received of many awards such as the Scientist Award in the senior category, the Certificate of Excellence for outstanding achievement and service to society. She has presented and published more than 55 papers in various International and national Conferences. The recent feather on her cap is acquiring of 'A' grade from National Assessment and Accreditation Council (NAAC) for MSRCASC for the 3rd consecutive time.

Dr. Aruna Karpur Dr. Aruna Karpur, a homeopath by qualification, has completed predictive homeopathy training in Mumbai. She is now working as a teacher at the school where Ameya and Aahana study. Dr. Aruna has been one of the key members of DART’s journey from the beginning. She is a major pillar of support to all DMD warriors and has always had a smiling and energetic presence at all of DART’s events.

From the DMD Warriors

Physiotherapy

Dr. S. Suresh Kumar


Byreddy Vishnu Vardhan Reddy Mr. Vishnu Vardhan Reddy is the Consultant Physiotherapist at DART specializing in Muscular Disorders. He obtained his Bachelor degree in Physiotherapy (B.P.T) from The Tamilnadu Dr.M.G.R Medical University in 1999. He works as a Senior Physiotherapist and Out-Patient Department coordinator at the Diagnostic & Research centre, Spastic Society of Karnataka. He also works as a consultant at Pratham Hospital, Kalyan Nagar, Bangalore, India.

Dr. S. Suresh Kumar is the Senior Physiotherapist at Mehta Multi-Speciality Hospital and the President of the Muscular Dystrophy Association India. He has authored a Chapter in Indian Academy of Paediatrics on Physiotherapy & Rehabilitation and for more than 20 years. He has been a Clinical Evaluator for NIH, USA and the European clinical trials especially CINRG/Treat NMD – muscle research groups.

Anands,Sheetys,Petkars,Karpurs,Chowhans,Dr.SarthakKamath,Pradeeps

Programme

www.dartindia.in RSVP:90008516322 [email protected]

09:57 am : Introduction by Master of ceremonies, Berty Ashley 10:00 am : Welcome Address by Mr. R.S. Anand, President DART 10:05 am : Guest Of Honour: Prof. Steve Wilton - Murdoch University "Current and Future Research in Genetic Therapy” 10:15 am : Key Note Speaker : Dr. Soumya Swaminathan - WHO “ Rare/Orphan/ Genetic disorders and the role of WHO on a Global scale” 10:25 am : Dr. Arun Shastry - Chief Scientific Officer - DART “Role of DART in pioneering research and therapy in India” 10:30 am : Dr. Meenakshi Bhat - Center for Human Genetics “Role of genetic counseling in Rare genetic disorders” 10:40 am : Prof. Vijay Chandru – Co-Founder Strand Life Sciences “Rare/Orphan Disorders and role of the government in treatment of the same”

Open forum for Discussion - 10 Minutes 11:00 am : Ms Manju Balasubramanyam – Principal, Delhi Public School Bangalore North ”The importance of Inclusive Educational Policies and Infrastructure” 11:10 am : Dr. A. Nagarathna – Principal, Ramaiah College “Role of Educational Institutions in the lives of special children” 11:20 am : Ms Rema NandaKumar - Dean of Samsidh Group of School “Role of Mentors in the development of character of special children” 11:30 am : Dr. Aruna Karpur – Teacher and DMD parent “Being a parent, mentor and friend for a special child” 11:35 am : Ms Jesse Jeffrey - Developmental Therapist “Tele-health as a game-changer for Children with Special Needs ”

Open forum for Discussion - 10 Minutes

Date: Monday, 7th September 2020 on Zoom Session1

Programme

Ms. Movin Anand Hitesh Chowhan Berty Ashley

www.dartindia.in RSVP:90008516322 [email protected]

Free Registration for the Conference : https://dartindia.in/wdad20/

11:50 am : Mr. Vishnu Vardhan Reddy - Spastic Society of Karnataka “ Active Physiotherapy in children with muscular disorders” 12:00 pm : Dr. S. Suresh - Kanchi Kamakoti Child Trust “ Importance of Physiotherapy in DMD Children”

Open forum for Discussion - 10 Minutes 12:10pm : DMD Warriors - “Our bravehearts and their parents, sharing the spirit of positivity” 12:25pm : Dr. IC Verma – Sir Gangaram Hospital “Role of Clinicians and Hospitals in Clinical Research/Therapies” 12.30pm : Dr. Ann Agnes Mathew - Sagar Hospital, Bangalore “Clinical Research/Therapies for Rare Muscular disorders” 12:35pm : Dr. Renu Suthar – PGIMER, Chandigarh “Deflazacort Vs.Prednisone – The debate continues” 12:40pm : Dr. Andoni Urtizberea - Hospital Marin & TREAT-NMD “Clinical Therapies in DMD and role of TREAT-NMD in the Global Therapy scene” 12:50pm : Dr. Sarthak Kamath – Psychiatrist and DMD Warrior “My journey over and above DMD’ 12:58pm : Vote of Thanks – Berty Ashley – Senior Scientist, DART

Open forum for Discussion

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OrganisingCommittee

Date: Monday, 7th September 2020 on Zoom Session2

OrganisingChairperson OrganisingSecretary JointSecretary

2nd dart international ( virtual ) conference -2020

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