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U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby’s muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage. The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called spinal muscular atrophy. The treatment targets a defective gene that weakens a child’s muscles so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year.
Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn’t work.
The one other medicine for the disease approved in the U.S. is a drug called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza’s maker, charges a list price of $750,000 for the first year and then $350,000 per year after that.
‘Dramatically transforms’ lives
The independent nonprofit group Institute for Clinical and Economic Review, which rates the value of expensive new medicines, calculated that the price of the new gene therapy is justifiable at a cost of $1.2 million to $2.1 million because it “dramatically transforms the lives of families affected by this devastating disease.”
ICER’s president, Dr. Steven D. Pearson, called the treatment’s price “a positive outcome for patients and the entire health system.” The defective gene that causes spinal muscular atrophy prevents the body from making enough of a protein that allows nerves that control movement to work normally. The nerves die off without the protein.
In the most common type, which is also the most severe, at least 90% of patients die by age 2, and any still alive need a ventilator to breathe. Children with less-severe types become disabled more slowly and can live for up to a couple decades.
Zolgensma works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.
In patient testing, babies with the most severe form of the disease who got Zolgensma within 6 months of birth had limited muscle problems. Those who got the treatment earliest did best. Babies given Zolgensma after six months stopped losing muscle control, but the medicine can’t reverse damage already done.
Evelyn Villarreal was one of the first children treated, at eight weeks. Her family, from Centreville, Va., had lost their first child to spinal muscular atrophy at 15 months. Two years later when Evelyn was born a test showed she also had the disease, so the family enrolled her in the gene therapy study at Nationwide Children’s Hospital in Columbus, Ohio.
Evelyn is now 4½ years old and showing no muscle problems other than minor trouble standing up, said her mother, Elena Villarreal. She has been feeding herself for a long time, she draws and speaks well, and will be starting kindergarten in the fall. “She’s very active and goes to the playground a lot,” said Elena Villarreal. “She’s walking and even jumping.”
It is too early to know how long the benefit of the treatment lasts, but doctors’ hopes are rising that they could last a lifetime, according to Dr. Jerry Mendell, a neurologist at Nationwide Children’s. Mendell led one of the early patient studies and is Evelyn’s doctor. “It’s beginning to look that way,” he said, because a few children treated who are now 4 or 5 still have no symptoms.
Early diagnosis is crucial, so Novartis has been working with states to get genetic testing for newborns required at birth. It expects most states will have that requirement by next year. The FDA said side effects included vomiting and potential liver damage, so patients must be monitored for the first few months after treatment. (VOA)
By Mohammed Shafeeq
Heart health of every person is in his hands and if people adopt good diet, healthy lifestyle, do some exercises and avoid smoking they can very well prevent a large number of heart diseases, says Cardiological Society of India (CSI) president Dr P.P. Mohanan.
As part of its efforts to create public awareness on how to prevent cardiovascular diseases, CSI at its 73rd conference in Hyderabad has released a video featuring India's former cricket captain Kapil Dev and a book about the common things in cardiology written by 150 cardiologists.
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For the first time, the CSI has roped in a celebrity to come out with a video, through which it is trying to project the importance of avoiding heart attack in youngsters.
"I hope it will be circulated in various forums so that people realise the importance of looking after their heart health, which is in their hands. If they adopt a good diet, lifestyle, do some good exercises and avoid smoking we can very well prevent a large number of heart diseases," the eminent cardiologist told IANS. He described the book as A to Z of cardiology.
"It's all about normal heart, heart diseases, how to prevent them and if you are unfortunately developing them how to treat them. The book is in English but I hope it will be translated in every possible language."
The CSI is adopting a two-pronged strategy -- public information and educating cardiologists. Stating that public awareness has been one of the fortes of CSI, he said they want to improve on it in every possible way.
With 5,000 members across the country, the CSI is working towards the prevention of cardiovascular diseases (CVD) and the eradication of cardiovascular mortality to raise awareness among people about cardiovascular diseases and nutritious diets. It is making efforts to increase awareness about the correlation between cardiovascular diseases and the environment and lifestyle.
The four-day conclave of cardiologists, which ended Sunday, discussed issues like clinical cardiology, preventive cardiology, interventional cardiology, imaging cardiology and Artificial Intelligence and digital technology in practice of cardiology.Unsplash
CSI's president elect Dr P.S. Banerjee told IANS that prevention goes side by side with awareness. The cardiologists' body will be approaching the government in reaching out to people in remote areas.
"We will write to the government. If it takes our help we can send our representatives to organise small meetings in local languages on what to do to prevent heart attack or any heart disease," he said.
The four-day conclave of cardiologists, which ended Sunday, discussed issues like clinical cardiology, preventive cardiology, interventional cardiology, imaging cardiology and Artificial Intelligence and digital technology in practice of cardiology.
Dr Banerjee, who was the scientific chairperson of CSI2021, pointed out that the whole subject of cardiovascular medicine was covered. Speakers selected for sessions reputed in their sub-specialties like heart failure, preventive cardiology hypertension and diabetes. There were also joint sessions with American College of Cardiology, European Society of cardiology, European Society of Heart Failure, European heart journal for exchange of views.
Quit smoking to prevent a large number of cardiovascular diseases.Unsplash
The cardiologists met after a gap of two years. Dr Mohanon described it as a fantastic meeting where thrust was given to innovations happening. "Cardiology is one field where we embrace whatever innovations are happening for the benefit of patients," said the CSI president.
"We have been trying to reach out to healthcare professionals, cardiologists, physicians, teach them newer innovations in cardiology, new guidelines for medical treatment and how best to utilise them for better care."
Dr Mohanan said CSI had been trying to assimilate new innovations. "This time we have given more importance to digitization. Covid has taught us a lot of newer things. We learnt a lot of things and we unlearned a lot of things. We are trying to learn about newer things, how to incorporate every new knowledge available world over and improve cardiac care," he said.
Realising the key role the government has in public education and awareness, the cardiologists' body is looking to influence the policymakers like its counterparts in the United States.
"We have to advise them about the importance of physical activity and healthy diet."Unsplash
"We have to influence policy makers just like we did for the smoking ban. We have to advise them about the importance of physical activity and healthy diet. Policy makers have a huge say. We will try to influence them like what other associations are doing. The American Heart Association has a huge influence on their government. The CSI will try to emulate that and come out with a solid proposal on how to live a good healthy life so that you prevent heart diseases," the CSI president said.
Dr Banerjee said they have to depend on government assistance as the task of reaching out to a big population, especially in rural areas, is huge. "We need government assistance as society alone can't do it. This requires a lot of money," he said.
He proposed to set up small groups of young doctors who will go to remote areas and with the help of local authorities organise seminars. They will give demonstrations on aspects like CPR, lifestyle modification, benefits of physical exercise, good diet, avoiding tobacco and alcohol. (IANS/SP)
(Keywords : heart, health, Kapil Dev, diet, lifestyle, exercises, awareness, cardiovascular, diseases, book, nutritious, prevention, physicians, Cardiological Society of India.)
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By Quaid NajmiMumbai
Set up in 1958 as the country's second IIT -- after IIT Kharagpur (IIT-KGP, 1950) -- in the list of 23 IITs currently, IIT-B enters the momentous diamond jubilee year of its historic first convocation held on December 22, 1962, when around 70 wide-eyed young men passed out of the institute.
Decades later, the golden jubilee convocation in 2012 was attended by then Prime Minister Manmohan Singh. The institute has a little more than 12,005 students in 2021, as per the current annual report released by its Director, Professor Subhasis Chaudhuri.
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The groundwork for the first IIT-KGP was inspired and prepared well before Independence by the late Bengal academician Humayun Kabir, who later served as the Education and Civil Aviation Minister of India.
After the country achieved freedom in 1947, its first Prime Minister Pandit Jawaharlal Nehru pushed the idea forward and even laid the foundation stone for IIT-B, according to the Council of Indian Institutes of Technology (CIITs).
Nehru wanted science and technology to play a prominent role in modernising the new India and meeting the needs of its growing population, and envisioned that the IIT system would, over time, produce scientists and technologists of the highest calibre who would engage in research, design and development to help build the nation towards self-reliance in her technological needs.
"The institutions were to be designed with the necessary dynamism, flexibility of organisation and capacity to adapt in the light of expanding knowledge and changes in the socio-economic requirements of a modern society," says the CIIT.
The earliest IITs got the benefit of material assistance and academic cooperation from certain developed countries -- IIT-B from USSR, IIT-Madras from Germany, IIT-Kanpur from the US and IIT-Delhi from the UK.
Over the years, the IITs have created world-class educational platforms dynamically sustained through internationally recognised research, based on excellent infrastructural facilities.Wikipedia
Over the years, the IITs have created world-class educational platforms dynamically sustained through internationally recognised research, based on excellent infrastructural facilities.
The faculty and alumni of IITs have made a huge impact in all sectors of the society, both in India and abroad, and the IITs are globally recognised as 'centres of academic excellence', reputed for the outstanding calibre of the students graduating from them.
Over the years, even IIT-B grew in leaps and bounds and now ranks nationally and globally among the most renowned institutes of excellence in various fields of technology.
Compared to the first girl -- Tejaswini Saraf (1966 batch) -- who turned heads at IIT-B, being the lone female student among 300 boys, today the situation is different with 20-25 per cent female students on the campus.
View from Boat House, Powai Lakewikipedia
As the President of the IIT-B Alumni Association (IITBAA), Deepak Patil, says, at IITs, the mind is trained not only academically, but also to think deeply, rationally, to handle any problem, to go to the root and evolve a logical solution.
IITBAA Chairman Girish Nayak says IIT education makes the student sharper and analytical, trains them overall to solve any kind of problems, grapple any challenges in life without getting surprised or overwhelmed, and this is something that stays with them forever.
The duo feels that the IITs offer an excellent opportunity for personality development, total independence, no family support systems to shield them from any emotional problems, thrown together with total strangers from different parts of the country and learn to live together.
"It's here that we realise that there are many who are not only as brainy as you, probably more intelligent than you... There would be hot debates of high intellectual levels on practically any topic under the sun, from technology to politics. All of these adds to your personality and intellect and yet makes you humble," said Patil.
View of IIT hostelsWikipedia
Plus, the students here get the advantages of an outstanding faculty, staff, academic-industry connection, and the rich experiences of the trail-blazer alumni, which result in achievements that make global headlines.
A few of the many notables who have passed out of IIT-B over the past six decades are: BSE MD & CEO Ashish Chauhan, Syntel founder Bharat Desai, Infosys Co-founder Nandan Nilekani, Twitter Inc. CEO Parag Agrawal, Senior Advocate Colin Gonsalves, ex-BMC Commissioner Jairaj Phatak, ex-Union minister Jairam Ramesh, late Goa CM Manohar Parrikar, mathematician Ravindran Kannan, ex-Dean of Harvard Business School Nitin Nohria, economist Ajit Ranade, and ex-President of Bell Labs Arun Netravali, among others. (IANS/SP)
(Keywords : IIT Bombay, IIT. geniuses, convocation, science, technology, knowledge, dynamism, calibre, education, intelligent, student, debate, faculty, advantages, outstanding.)
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With the inauguration of Kashi Vishwanath corridor in Varanasi on December 13, the whole of Uttar Pradesh will be lit up similar to Diwali-like celebrations. This year the people of the state will invoke Lord Shiva in their homes by lighting lamps on the occasion of the inauguration of Kashi Vishwanath corridor, the highest revered shrine of Hindu faith. Along with this, a laser show and fireworks will light up all temples, streets, 'crossroads' and other public places in Varanasi.
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The planners will celebrate the festival of 'Deepotsav' on the lines of the 'Grand Kashi, Divya Kashi' in the whole of Uttar Pradesh. For this every BJP worker will light a lamp in the village, town and city. The BJP believes that this will create a festive atmosphere with ideological commitment which will send out the message of strong cultural ethos.
After the inauguration on December 13 in the evening, the Prime Minister will take a tour of the Ganga river in a boat and perform the 'Ganga aarti'. During this tour, the Chief Ministers of various BJP-ruled states will also be present. The Prime Minister will hold talks with the Chief Ministers and public representatives the very next day. After this, Modi will visit the Swarved temple, Umrah, and a CNG plant in Shahanshahpur and leave for Delhi on December 14 in the afternoon.
Also Read : The Varanasi Hot Air Balloon Festival
On the day of the inauguration of Kashi Vishwanath corridor, all boats will be decorated with colorful lights which will be the main centre of attraction. An appeal has been made by the state administration to all the people to light lamps in their homes on the lines of Diwali celebrations. The people have been instructed to keep the streets and their locality clean and cooperate in the public cleanliness drive.
Diyas will be lit in every house, every temple, streets and BJP offices in the state. Unsplash
If political experts are to be believed, amid the ongoing Ram temple construction in Ayodhya, the BJP is once again trying to assert power through its ideological plank of Hindutva and its cultural agenda, while raising issues like building of a temple in Mathura.
A senior BJP leader involved in formulating the election strategy in Uttar Pradesh and working closely with the party's central leadership said that once again the party is going to celebrate Deepotsav on December 13.
Several BJP workers have been asked to make the occasion a grand success. BJP Kashi regional President, Mahesh Chandra Srivastava said, "On December 13, there will be a festive atmosphere in the state. Diyas will be lit in every house, every temple, streets and BJP offices in the state. We are going to celebrate a grand festival."
Kashi Vishwanath Dham, the work of which is in final stages, is the dream project of Prime Minister Modi. The corridor is now gleaming with lights. After the launch of the corridor, Chalo Kashi month' will also begin under which many programmes will be organised here.
After the inauguration on December 13 in the evening, the Prime Minister will take a tour of the Ganga river in a boat and perform the 'Ganga aarti'.Unsplash
According to the information received from the state government, a symbol of nationalism will be visible between Ganga and Lord Vishwanath temple which will give a new identity to Kashi. Idols of Rani Ahilyabai, Mother India (Bharat Mata), Kartikeya and Adi Shankaracharya will be installed in the Dham.
Kashi Vishwanath Dham is spread over an area of 54,000 square metre. The construction work of the Dham is being completed in two phases. The work of the first phase is in the final stage which includes construction of Mandir Chowk, Varanasi city gallery, museum, multipurpose auditorium, hall, devotee facilitation centres, public facility, spiritual house, Godoulia gate, 'Bhogshala', shelter for priests and sewadars, spiritual book palace, among others.
The statue of Mother India, a symbol of nationality, will also be seen between the Lord's court and the sacred Ganga. The idol of Kartikeya and Adi Shankaracharya will be installed in the grand courtyard of Kashi Vishwanath Dham. The statue of Queen Ahilyabai, who renovated the Kashi Vishwanath temple in 1669 will also be installed here. (IANS/SP)
(Keywords : Uttar Pradesh, diwali, Kashi Vishwanath, Varanasi, December, Prime Minister, Narendra Modi, ganga, BJP, corridor, nationalism, people.)