Denis Costello - Cambridge Rare Disease Summit 2015
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Denis Costello, Rare Connect, EURORDIS, spoke in the 'New Initiatives on the Horizon' panel at the Cambridge Rare Disease Summit 2015.
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This document discusses empowering patients to take a more active role in drug discovery and development. It notes that for many rare diseases, the science is incomplete and drug development is slow. However, patient organizations are already leading many scientific and medical advances. The document advocates for a more collaborative approach where patients work with researchers and companies. It presents examples where patient groups have helped identify new patients, generated data to help trials, and invested in research. It argues that patients need more access to tools and technologies to drive diagnosis and drug development themselves for ultra-rare diseases. An envisioned "23andMe of drug development" could help facilitate this.
Natalie Rebeyev rare disease nurse network crdn summit 2017
The document discusses the potential for a telemedicine pilot program to serve patients with rare diseases. It summarizes surveys that found over 50% of patients would be receptive to telemedicine and over 90% are willing to learn video call applications. The document estimates a 3-year pilot program could save £6.8 million in healthcare costs while costing approximately £975,000 to implement. Key operational requirements for the pilot include videoconferencing capabilities and secure data protection.
Daniel lewi cats foundation crdn summit 2017
The document summarizes the history and work of a charity focused on Tay-Sachs and Sandhoff diseases. It describes how the charity was formed in 2011 in response to a research team's need for funding and patient identification support. The charity provides equipment, respite trips, and other support to families, and works closely with researchers conducting a gene therapy trial for the diseases. Its goals are to complete the current trial and eventually develop treatments that could eliminate the need for the charity.
The cats foundation power voices dan lewi - crdn summit 2016
The CATS Foundation was founded in 2011 to support families affected by Tay-Sachs and Sandhoff diseases. It provides equipment, respite trips, and a support network for these families. The foundation also supports research for potential treatments for these currently terminal illnesses that cause children to lose abilities like walking, speaking, and hearing. Currently, the foundation is working with Professor Cox and is close to starting a clinical trial for an potential enzyme replacement therapy treatment.
Rare revolution the voice of rare crdn summit 2016
Teddington Trust is encouraging people to join the #RareRevolution by tapping into the talent, creativity, and passion of the rare disease community through patient education, peer education, and raising awareness. The document discusses the rare diseases of the author's father and nephew to inspire others that with preparation and belief, achieving your goals is possible, even when facing a rare condition. It directs people to the Rare Revolution magazine website and social media accounts to get involved.
Lucy mc kay crdn 2016
Students 4 Rare Diseases is an organization founded by Dr. Lucy McKay that aims to educate medical students and future doctors about rare diseases. It coordinates with 33 medical schools in the UK, which have almost 7,000 medical student places each year. The organization's educational coordinator works to keep its website and social media updated, contacts medical schools consistently, and organizes an annual symposium, with the goal of ensuring future doctors are prepared to consider and diagnose rare diseases.
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There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
In addition to infrastructure and capacity constraints, CAR-Ts face a very different risk-benefit dynamic in autoimmune compared to oncology, highlighting the need for tolerable therapies with low adverse event risk. CAR-NK and Treg-based therapies are also being developed in certain autoimmune disorders and may demonstrate favorable safety profiles. Several novel non-cell therapies such as bispecific antibodies, nanobodies, and RNAi drugs, may also offer future alternative competitive solutions with variable value propositions.
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Basavarajeeyam - Ayurvedic heritage book of Andhra pradesh
Basavarajeeyam is an important text for ayurvedic physician belonging to andhra pradehs. It is a popular compendium in various parts of our country as well as in andhra pradesh. The content of the text was presented in sanskrit and telugu language (Bilingual). One of the most famous book in ayurvedic pharmaceutics and therapeutics. This book contains 25 chapters called as prakaranas. Many rasaoushadis were explained, pioneer of dhatu druti, nadi pareeksha, mutra pareeksha etc. Belongs to the period of 15-16 century. New diseases like upadamsha, phiranga rogas are explained.
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Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar lead (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
6. Describe the flow of current around the heart during the cardiac cycle
7. Discuss the placement and polarity of the leads of electrocardiograph
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1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. Chapter 3, Cardiology Explained, https://www.ncbi.nlm.nih.gov/books/NBK2214/
7. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
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Estas diretrizes consolidadas apresentam algumas recomendações novas e já bem fundamentadas sobre cuidados pós-natais de rotina para mulheres e neonatos que recebem cuidados no pós-parto em unidades de saúde ou na comunidade, independentemente dos recursos disponíveis.
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O estabelecimento da amamentação e o manejo das principais intercorrências é contemplada.
Recomendamos muito.
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Esta publicação só está disponível em inglês até o momento.
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Denis Costello - Cambridge Rare Disease Summit 2015
- 1. New Initiatives on the Horizon Denis Costello, EURORDIS Cambridge Rare Disease Summit – September 14, 2015
- 2. Key indicators • 77 rare disease communities, most recently Stiff Person Syndrome • 269 volunteer moderators • 660 rare disease patient group partners • 60,000 visitors/month
- 3. New Logo
- 4. New Homepage
- 5. New Homepage
- 7. Visit: https://www.rareconnect.org/topics (leave your email for notification of launch) Overview • Ask questions of the Rare Disease community on any related topic (disease, social questions, advocacy) • Answer questions • Write blog or tell story • Become a moderator Discussion Groups
- 10. • Specialised Social Services • RD National Plans • Rare Diseases International • Patient engagement in Drug Dev. • Summer School Alumni • Crowdfunding • European Reference Networks + Centres of Expertise Featured Discussion Groups
- 11. • Currently localised in 5 languages (EN, FR, ES, IT, DE) with 2 pass translation (Google Translate + Human Translation) • 2015: launch of Portuguese (June) and Russian (November) • 2016: Working with EURORDIS National Alliances to translate into more EU languages Language Roadmap