Podcasts about zolgensma

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The CDC has rescheduled a vaccine meeting for April, while President Trump is considering Texas Republican Michael Burgess as the new director of the agency. J&J's Tremfya has won approval for expansion in treating Crohn's disease, while Adaptimmune is facing financial uncertainty despite sales of its T cell therapy. Alnylam's Amvuttra has been approved as the first RNAi silencer for a rare type of cardiomyopathy, setting up competition with Pfizer and BridgeBio. Sino Biological has developed reagents for the 2025-2026 influenza vaccine strains. In other news, J&J plans to boost US manufacturing following tariff threats, Novartis' Fabhata has been approved as the first therapy for a rare kidney disease, and Sanofi commits up to $1.9 billion for Dren Bio's bispecific antibody for autoimmune diseases. Paratek has acquired Optinose for up to $330 million, Purdue Pharma has filed for bankruptcy again to support an opioid settlement, and Novartis' Zolgensma has been found effective in older children.The FDA has approved Alnylam's Amvuttra as the first RNAi silencer for a rare type of cardiovascular disease called ATTR-CM, following the approval of BridgeBio's Attruzy for the same condition. This approval has sparked a three-way race in the rapidly expanding space, with Pfizer's Tafamidis also in the competition. Alnylam is optimistic about Amvuttra's unique mechanism of action, which targets the disease at its source by rapidly reducing the disease-causing TTR protein. This approval comes after Attruzy was also approved for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in June 2022. This development has brought cardiovascular disease back into the spotlight, with other companies launching new drugs and treatments for various heart conditions.

Hoje, ‘No Pé do Ouvido, com Yasmim Restum, você encontra essas e outras notícias: Consignado CLT já tem 40 milhões de simulações e 11 milhões de contratos. Netflix aposta em estratégia mais focada para virar referência nos games. Morre George Foreman aos 76 anos. CNJ estuda adotar biometria para reduzir erros em mandados de prisão. Premiê do Canadá convoca eleições antecipadas à sombra da guerra comercial. Branca de Neve: o maior fracasso de estreia dos live-actions da Disney. Zolgensma: remédio de R$ 7 milhões para tratamento da AME será oferecido no SUS e Papa Francisco recebe alta após 38 dias de internação.See omnystudio.com/listener for privacy information.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sanofi has committed up to $1.9 billion to acquire Dren Bio's bispecific antibody for autoimmune disease, adding to its investments in the immunology portfolio. The deal comes after the tragic death of a patient who had taken the gene therapy Elevydys, prompting the Duchenne patient community to vow to push on. Paratek Pharmaceuticals has acquired Optinose for up to $330 million, while Senate Democrats demand the return of fired CDC staff. Sino Biological has developed recombinant antigens for the 2025-2026 influenza vaccine strains, and Purdue has filed for bankruptcy to support a $7.4 billion opioid settlement. Doctors continue to rally behind vaccines amidst doubts and misinformation, and Novartis' intrathecal Zolgensma has shown effectiveness in older children. TC Biopharm and Cargo have enacted steep workforce reductions. Pharmaceutical companies are also preparing for upcoming events, including webinars on AI regulation and drug development. Job opportunities in the pharmaceutical industry are available at companies like Takeda, Eli Lilly and Company, and Novo Nordisk.

In this episode of Molecule to Market, you'll go inside the outsourcing space of the global drug development sector with Imran Kausar, Founder of DeepHealth Consulting. Your host, Raman Sehgal, discusses the pharmaceutical and biotechnology supply chain with Imran, covering: His unusual blend of experience led to a road into big pharma... Switching the security of Big Pharma to join an exciting, scrappy, start-up biotech... before an exit to Roche Being part of the gene therapies evolution with an innovative biotech that later became Novartis Gene Therapies His views on the thorny issue of CGT products' price (and value) Opting to start his own business aimed at supporting the future of healthcare Imran Kausar is a visionary healthcare and life sciences leader, blending medical expertise with strategic foresight. A medical doctor by training, Imran specialized in anesthesia and critical care before transitioning to the pharmaceutical industry. Having launched five rare disease medicines and spearheaded innovative work in gene therapies like Zolgensma and Luxturna across Europe, Imran has earned his reputation as a trailblazer in the industry. As the founder of DeepHealth Consulting, Imran leads the flagship program DeepHealth Futures, which equips life sciences teams to navigate the transformative forces of AI, longevity science, and precision medicine.With nearly two decades of experience in medicine, medical affairs, and general management, Imran's career spans the cutting edge of rare diseases, antisense oligonucleotides, and healthcare transformation.    Please subscribe, tell your industry colleagues and join us in celebrating and promoting the value and importance of the global life science outsourcing space. We'd also appreciate a positive rating! Molecule to Market is also sponsored and funded by ramarketing, an international marketing, design, digital and content agency helping companies differentiate, get noticed and grow in life sciences.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Medtronic's chief medical officer of acute care and monitoring has departed for a new role. The FDA has finalized voluntary malfunction summary reporting guidance. Penumbra is laying off 71 people after axing its virtual reality division. Qiagen and AstraZeneca have expanded their companion diagnostic pact. The Association for Molecular Pathology is suing to block an FDA lab test rule. Layoffs in the medical device industry continue to be a trend. Industry news includes the launch of the first over-the-counter glucose monitor, unexpected partnerships in diabetes tech, and pushback on FDA regulation of lab-developed tests.The text discusses various updates in the biopharma industry, including Alnylam's heart drug data sparking debate, Novo building a heart failure case for semaglutide, and a slump in cell and gene therapy investment. The industry is shifting towards more patient-centric commercialization strategies, with companies like Pfizer and Lilly breaking into the direct-to-consumer market. The summer has been a mixed bag for biotech, with market fluctuations and limited IPOs and acquisitions. Alnylam's full study data on its heart drug vutrisiran showed benefits but also raised skepticism. Novo's analysis showed semaglutide's effectiveness in reducing heart failure risk. Additionally, PBM executives are facing fines for alleged perjury in a house hearing defending their business practices.The text discusses the pricing and sales of gene therapies, particularly focusing on Novartis' Zolgensma, which has been the only gene therapy to cross the blockbuster threshold with sales of $1.2 billion in 2021 and $1.4 billion in 2022. While high price tags have not always resulted in significant returns for pharmaceutical companies with gene therapies, Zolgensma has been an exception. Other companies have faced challenges with new gene therapies, such as Biomarin Pharmaceuticals, which recently announced layoffs due to dismal sales of its hemophilia A gene therapy Roctavian.Alnylam Pharmaceuticals has released detailed data on their drug Vutrisiran, showing its benefits in treating a progressive and fatal heart condition known as ATTR cardiomyopathy. The results were published in the New England Journal of Medicine. While the data confirms the therapy's benefit, there are still questions about how exactly Vutrisiran should be used in treating the disease. The study is expected to continue to fuel debate among doctors.The text discusses the recent slump in investment in cell and gene therapy, with fewer venture funding rounds closed by developers in the first six months of 2024. Two companies, Biomarin and Tome Biosciences, are cutting jobs, with over 200 and over 100 layoffs respectively. Biomarin has made changes to its executive team and drug pipeline to refocus resources. Additionally, Bayer has partnered with RNA drugmaker Nextrna Therapeutics to develop new cancer therapies. The text also highlights the challenges faced in clinical trials, with nearly 80% failing to meet enrollment goals and schedules. The importance of understanding social determinants of health for research is emphasized.Biopharma Dive's Gene Therapy Weekly provides news and insights on gene therapy for biopharma leaders.

A juíza de instrução considerou que Marcelo Rebelo de Sousa não teve um comportamento “neutro” no caso das gémeas luso-brasileiras e, por isso, defendeu que o Presidente da República deveria ter sido investigado. A mãe das gémeas tratadas com o medicamento Zolgensma é ouvida hoje na comissão parlamentar de inquérito. Neste episódio, conversamos com os jornalistas Rui Gustavo e Hugo Franco.See omnystudio.com/listener for privacy information.

The work of gene therapy pioneer Genethon, a non-profit organization created by the patient association AFM-Telethon, has already seen its research lead to Zolgensma, the gene therapy for spinal muscular atrophy, as well as a growing pipeline of candidates for other rare diseases. But it came to realize it needed to pursue more than just licensing agreements to ensure its work ultimately benefited people who needed its therapies. As a result, the organization has taken a range of different approaches to ensure the development of its gene therapies. We spoke to Frederic Revah, CEO of Genethon, about the limits of licensing out its discoveries to biopharma, the different development strategies it pursues, and how it determines the best path for a particular development program.

A Inspecção-Geral da Saúde deu a conhecer o relatório onde é admitido que o Governo, o Hospital de Santa Maria e o Infarmed agiram de forma irregular e beneficiaram as gémeas luso-brasileiras no acesso à terapêutica com o medicamento Zolgensma, com custo por doente na ordem dos dois milhões de euros. No mesmo relatório é dito que o Palácio de Belém procurou condicionar a investigação. O Chega anunciou que vai pedir a criação de uma Comissão Parlamentar de Inquérito. Neste episódio, conversamos com Rita Ferreira, editora de Sociedade do jornal Expresso.See omnystudio.com/listener for privacy information.

A conclusão da Inspecção-Geral das Actividades em Saúde (IGAS) é clara: "Não foram cumpridos os requisitos de legalidade no acesso das duas crianças a consulta de neuropediatria" do Hospital de Santa Maria, em Lisboa, por parte das gémeas luso-brasileiras que foram tratadas com o medicamento Zolgensma — na altura, um dos mais caros do mundo —para a atrofia muscular espinhal. Como fica a Presidência da República depois de se saber que dificultou a investigação?See omnystudio.com/listener for privacy information.

O Ministério Público investiga possível ligação do chefe de Estado para acelerar o tratamento de gémeos brasileiros, cuja família é amiga de seu filho, residente em São Paulo, com Zolgensma, um dos medicamentos mais caros do mundo. É administrada uma dose única, que custa dois milhões de euros.

今年八月，健保將納入全球最貴的藥物之一 注射一劑費用高達4900萬元的「諾健生（Zolgensma）」 為何該藥價格如此驚人？又為何健保選擇納入健保用藥？

Video verzia MEDI NOVÍN: https://bit.ly/45ZTcI2V dnešných MEDI novinách sa budeme venovať aj týmto témam:dvojmiliónový liek Zolgensma by mohol byť zaradený do zoznamu liekov preplácaných z verejného zdravotného poistenia,ministerstvo zdravotníctva SR zverejnilo zoznam nemocníc, ktoré splnili podmienky na získanie finančných prostriedkov z plánu obnovy,novým dekanom Lekárskej fakulty Slovenskej zdravotníckej univerzity v Bratislave bude gastroenterológ Ladislav KuželaPre C4P, s.r.o. pripravila SITA - Slovenská tlačová agentúra.

Video verzia MEDI NOVÍN: https://bit.ly/45ZTcI2V dnešných MEDI novinách sa budeme venovať aj týmto témam:dvojmiliónový liek Zolgensma by mohol byť zaradený do zoznamu liekov preplácaných z verejného zdravotného poistenia,ministerstvo zdravotníctva SR zverejnilo zoznam nemocníc, ktoré splnili podmienky na získanie finančných prostriedkov z plánu obnovy,novým dekanom Lekárskej fakulty Slovenskej zdravotníckej univerzity v Bratislave bude gastroenterológ Ladislav KuželaPre C4P, s.r.o. pripravila SITA - Slovenská tlačová agentúra.

Una semana más, Eva Belmonte nos lleva en su nave de los misterios para mostrarnos la verdad oculta tras el precio de los medicamentos. Hablamos del Luxturna o el Zolgensma, que puede llegar a costar más de un millón de euros por dosis.

As we take steps to improve ourselves during this glorious month of Elul, we need to remind ourselves of the value of every single improvement we make, no matter how small it may seem. Whatever we do is very precious to Hashem and will come back to benefit us at some point in the future. A young rabbi told, his daughter was diagnosed in 2019 with a disease called SMA. It is a life-threatening disease and at that time there was no known cure. The doctor told the young rabbi that the FDA just approved a new medication called Zolgensma, but the cost of it was a staggering $2.2 million. Furthermore, it was only approved for children under the age of two and his daughter was just a month shy of her second birthday. They immediately went through their insurance to get the medication but it was denied. They hired a lawyer to fight the insurance company on their behalf, but they lost the case. With just a week before her second birthday, they were at a standstill. With the young rabbi's daughter's life on the line, he had to come up with a plan and fast. He asked the doctor if they could just pay for the medication out of pocket. The doctor said of course, but how would they get the money to do it? The rabbi told the doctor he had a very rich father…and then said his father is Hashem who could do anything. The next day, they launched a chesed campaign online. And amazingly, in just five days, they collected the full $2.2 million. Ashrechem Yisrael ! However, it was not going to be possible to actually get the money for at least another few days, but they didn't have that kind of time. When a certain wealthy man heard about the urgency of the situation, he offered to lay out the $2.2 million from his own pocket. They took the money and managed to get the medication and have it administered in the hospital on the same day, the last possible day, the day before his daughter's birthday. It was July 19, 2019. And baruch Hashem, today the girl is six years old and doing great. When they went to pay back that man who laid out the money, he refused to take it. He said it was his zechut to give it and he didn't want the money back. The rabbi was blown away at the generosity of this man. Then they had to inform the more than 20,000 donors on the chesed page that they did not need their money anymore, but most of the people said they still wanted to keep the donation anyway, and so the rabbi used the over $2 million he had and started a new chesed organization that is helping people today on a daily basis. Our people are amazing! Their desire to help others knows no bounds. When the young rabbi had a chance to reflect and contemplate all the events that had taken place, he came to a striking revelation. Eight years before this when he was not yet religious, he had a very big test on his level. His friends were going out to party on the night of Tisha B'Av and they were urging him to come along. Deep down, he knew how wrong that was and managed to fight the peer pressure and stay home instead. He said that night was the turning point in his life. From that, he got the strength to grow in religion until eventually he became a rabbi. That night of Tisha B'av when he had that test was July 19, the exact same date that he almost miraculously was able to get that medication and give it to his daughter and save her life. Everything we do for Hashem is precious to Him. He remembers everything and uses it to help us. Everyone has their own tests on their own level. The common denominator between everyone is, nothing is ever considered small by Hashem from what they do. The sacrifices we make to do His will will be used to help us both in this world and the Next.

A pequena Ana Laura Orsi Batista, a Laurinha, foi diagnosticada com Atrofia Muscular Espinhal (AME) tipo 1 em março de 2020 e durante quase um ano inúmeras ações foram feitas para arrecadar os valores necessários para a campanha ‘Salve a Laurinha'. O movimento arrecadou o valor suficiente para a compra do medicamento Zolgensma, necessário para o tratamento e que é considerado o remédio mais caro do mundo. O repórter Nei Bordignon, conversou com Ana Paula Orsi, mãe da Laurinha, que contou como está a evolução logo após ela receber a dose do medicamento que mudou completamente sua vida. “Ela está bem, vem evoluindo a cada dia que passa. São muitos e muitos ganhos de um ano pra cá, mas de 6 meses pra cá é que a gente pode observar a evolução. É algo incrível de tudo o que a gente está acompanhando. Até em consulta com o neuropediatra dela, lá em São Paulo, ele também está impressionado do quanto ela evoluiu nos últimos meses”, comentou Ana Paula. Ouça abaixo íntegra da entrevista:

Rare Disease Day is celebrated every year on the 28th of February (or 29th in leap years) - the rarest day of the year. The day aims to raise awareness about rare diseases and the challenges faced by people living with these conditions. Rare diseases are the ones that affect a small percentage of the population, often fewer than 1 in 2,000 people. Muhammad Athif Fahri is one of those affected by a rare disease - he is suffering from Spinal Muscular Atrophy (SMA) Type 1, which affects his muscles and ability to breathe. Doctors have said the disease is incurable, and that without proper treatment, he will not make it past the age of two. We speak to Baby Athif's parents, Nur Azizah Zamri and Mohd Farhan bin Mohd Sait, about how life has changed for them all since Athif's diagnosis, and they also discuss how they are trying to get the right treatment for their youngest son, to save his life.*Zolgensma therapy is a treatment that can halt or even reverse the effects of the disease. Baby Athif needs to raise RM9 million to cover the cost of the treatment and related expenses. Find out how you can support him by clicking here and here.Image Credit: Nur Azizah Zamri & Mohd Farhan bin Mohd Sait

Cell and gene therapies are at the tip of everyone's tongues in biopharma. Significant headway has been made in this space, though there is more research that needs to be done and many more therapeutic areas to eventually apply this method of treatment to. Chris Fox, president of Novartis Gene Therapies, shares insights into her work with gene therapies and discusses commercialization challenges. Through its gene therapies business, Novartis builds on the capabilities created through its experience with Zolgensma, a gene therapy for spinal muscular atrophy. Fox also dives into how she entered the pharma space, her role at Novartis, balancing work and home life, and what the Novartis team is most proud of from 2022 as well as what they are most excited about in 2023.

Crônicas do cotidiano apresentadas pelo comunicólogo Raul Canal.

Conhecido como o “remédio mais caro do mundo”, o Zolgensma, que custa cerca de R$ 6 milhões, foi incorporado na lista de medicamentos distribuídos gratuitamente pelo Sistema Único de Saúde (SUS). O remédio é usado no tratamento de crianças com Atrofia Muscular Espinhal (AME), e deverá estar disponível na rede pública em até 180 dias, prazo estipulado pelo Ministério da Saúde. Seja por uma doença genética rara ou por outra condição clínica, como determinados tipos de câncer, muitos pacientes precisam de medicamentos extremamente caros e até difíceis de encontrar. E nesses casos, como o paciente pode obter os remédios de alto custo pelo sistema público de saúde? Há uma maneira de facilitar esse acesso? O direito é válido para todos? E quais são os critérios? Celso Freitas e o repórter Fábio Menegatti conversam com o advogado especialista em direito médico, Washington Fonseca.

This week on "The Top Line," we'll share highlights from the Cell & Gene Therapy Forum. Lack of access and multimillion-dollar costs have stirred debate over the appropriate price of potentially lifesaving cell and gene therapies. Fierce's Max Bayer spoke with a panel of stakeholders at the forum. They discussed the state of play for cell and gene therapies and how they can become a genuine game changer for the many, not the few. We also talk with executives from Poseida Therapeutics and AviadoBio.  To learn more about topics in this episode: Zolgensma for dementia? AviadoBio gains orphan tag for one-dose gene therapy  Poseida taps former Novartis VP as president of gene therapy  Fierce Biotech Summit: Disruptive gene technology is here—and society seems willing to pay for it Fierce Biotech Cell & Gene Forum "The Top Line" is produced by senior multimedia producer Teresa Carey with managing editor Querida Anderson and senior editors Annalee Armstrong, Ben Adams, Conor Hale and Eric Sagonowsky. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

El tratamiento a personas con enfermedades catastróficas, raras y huérfanas volvió al debate en el Pleno de la Asamblea Nacional. Tras escuchar los testimonios de padres cuyos hijos padecen atrofia muscular espinal (AME) y que no consiguen medicamentos, uno de los más caros del mundo, el Legislativo exhortó al presidente de la República, Guillermo Lasso, a que cumpla la Constitución y aplique las medidas respectivas para dotar a los servicios de salud de los medicamentos y tratamientos necesarios para brindar atención oportuna. También requirió que el mandatario disponga al Ministerio de Finanzas que asigne el presupuesto o genere los convenios o instrumentos internacionales necesarios, para lograr que el medicamento Zolgensma pueda llegar de manera oportuna a las familias ecuatorianas que padecen de atrofia muscular espinal. Esta resolución se incluirá en el Informe de actualización respecto de la vigencia de la situación de escasez de medicamentos necesarios para el tratamiento de las personas con discapacidad, y con enfermedades catastróficas, raras o huérfanas, así́ como la atención hospitalaria y el abastecimiento del cuadro nacional de medicamentos básicos en el Sistema Nacional de Salud. Laura Varela Torres, madre de Theo Tabango, de 7 meses de edad, con diagnóstico de atrofia muscular espinal (AME) tipo 1, expuso de manera desesperada y angustiada la enfermedad y los procesos que ha tenido que pasar, a fin de salvar la vida de su hijo. Pidió la intervención inmediata de las autoridades en la compra del medicamento, cuyo costo es de alrededor de dos millones de dólares. La asambleísta Yeseña Guamaní, proponente de la resolución, presentó su solidaridad e indicó que la enfermedad de Theo afecta la capacidad motriz, de ahí que es necesario que el Gobierno Nacional asuma su responsabilidad con los pacientes con enfermedades crónicas, catastróficas, raras y huérfanas. “Es una enfermedad que afecta la capacidad que tienen los niños para caminar, sentarse y controlar incluso los movimientos de su cabeza. De su lado, Gladys Tapia portavoz de niños con AME y madre de una niña con la enfermedad solicitó que se les permita el acceso a medicamentos, para que el tratamiento sea administrado a tiempo y que los tres tipos de tratamientos que existen para esta enfermedad sean ejecutados de manera oportuna. Advirtió que Ecuador es el único país en Latinoamérica que no ha firmado ningún acuerdo con laboratorios o instancias internacionales que les permita acceder a este tratamiento. Félix Galarza, presidente de la Federación Ecuatoriana de Enfermedades Raras, instó a las autoridades para que asignen un presupuesto permanente para proveer los medicamentos necesarios para el tratamiento. Norma Noguera, presidenta de la Fundación de Padres de Niños con AME, pidió concretar acuerdos para evitar que más niños mueran por falta de medicamentos, tras informar que en lo que va del año, siete niños han perdido la vida esperando el tratamiento que les corresponde por derecho. La primera vicepresidenta de la Legislatura, Marcela Holguín presentó el respaldo de la Asamblea a las personas que están en riesgo su vida a consecuencia de las enfermedades catastróficas, raras y huérfanas y que requieren de manera urgente la atención del Gobierno Nacional, para asegurar su tratamiento y la provisión de los medicamentos para su recuperación. --- Send in a voice message: https://anchor.fm/hechosecuador/message

Host: Jennifer Caudle, DO Guest: Sandra P. Reyna, MD Spinal muscular atrophy (SMA) is a challenging rare disease that requires a diagnosis and access to treatment as early as possible. To help us understand the importance of early treatment, Dr. Sandra Reyna from Novartis Gene Therapies shares key considerations for optimizing outcomes and gives us some insight into available SMA treatment options, focusing on the gene therapy ZOLGENSMA. © 2022 Novartis Gene Therapies, Inc.US-ZOL-22-0129 08/22

With 20+ gene therapy targets in Novartis Gene Therapies' pipeline, President Chris Fox and her team are hyper-focused on newborn screenings as the pathway to helping more and more patients. The company's initial gene therapy, Zolgensma, for spinal muscular atrophy (SMA) has been approved in more than 40 regions and countries and has been used to treat more than 2,300 patients worldwide. Fox details what's next on the commercialization front for the therapy, and she shares advice with Cell & Gene: The Podcast listeners about what it takes to commercialize a therapy.

Novartis has recorded two deaths after treatment with its gene therapy Zolgensma. The treatment is for spinal muscular atrophy. Two children in Russia and Kazakhstan died about five to six weeks after receiving Zolgensma. Both patients died of acute liver failure, a known side effect of Zolgensma. In today's episode, we will discuss these deaths and the gene therapy's safety. AstraZeneca may be a major oncology player, but the company has been noticeably absent in the first wave of CAR-T therapies. All that may be about to change. We chat with Dave Fredrickson, from the Big Pharma's oncology unit, about how the company is working on the next generation of these cancer therapies. To learn more about the topics in this episode:  2 deaths after Novartis' Zolgensma put gene therapy's liver safety in the spotlight once again  Late to the CAR-T game, AstraZeneca quietly works on its own off-the-shelf therapies  UPDATED: Bluebird bio's $2.8M gene therapy Zynteglo wins FDA backing. Will its US launch take flight? Hear ye, hear ye: FDA finalizes OTC hearing aid rule to amp up access, drive down costs  Sanofi scraps oral SERD strategy after 2nd failure in breast cancer  Are SERDs dead? 'Good question,' says Sanofi R&D chief, after French pharma's late-stage pipeline takes a beating  After billions in legal costs, Johnson & Johnson plans to halt talc sales worldwide AstraZeneca heads to court to contest former exec's move to crosstown rival GSK: report CPAP machine competitor ResMed nears $3.6B revenue in first year of Philips' ventilator recall  Merck bets big on circular RNA, paying $150M and dangling $3.5B in biobucks to work with Orna  AZ, Daiichi Sankyo's Enhertu breaks more ground, nabbing fast FDA nod in HER2-mutant lung cancer  We're looking for 2022's Fiercest Women in Life Sciences  The Top Line is produced by senior multimedia producer Teresa Carey with managing editor Querida Anderson and senior editors Annalee Armstrong, Ben Adams, Conor Hale and Eric Sagonowsky. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

O remédio mais caro do mundo

Frau Müller arbeitet im Bereich Arzneimittel der Krankenversicherung. Hier ist sie oft mit schlimmen Krankheiten von Menschen konfrontiert. In einer schwierigen Zeit ist sie dann oft auch einer der ersten Ansprechpartner und kann Sorgen nehmen. Wie sie es schafft, mit den verschiedenen Schicksalen umzugehen und wie das teuerste Medikament der Welt Leben retten kann, das erzählt sie in dieser Folge von Backstage Storys. Rückmeldungen gerne an podcast@debeka.de

En agosto de 2020 Marcela Márquez y Álvaro Leiva, padres del iquiqueño de tan sólo tres meses, Lucas Leiva Márquez, iniciaron una dura lucha por mantener con vida a su hijo tras ser diagnosticado con atrofia muscular espinal (AME) tipo 1, cuyo tratamiento es uno de los más costosos del mundo, alrededor de 1.560 millones de pesos. En abril de 2021 contra todo pronóstico lograron reunir el dinero para que Lucas recibiera el Zolgensma. El 19 de Abril de 2022 Lucas cumplió dos años y sus padres manifiestan su alegría por tener a su hijo, pero a la vez realizan un emotivo relato de este doloroso proceso que han vivido como familia.

Le prix des médicaments n'est pas fixé de la même manière d'un pays à un autre. Pour les plus chers, les entreprises pharmaceutiques justifient ces prix en raison des coûts exorbitants de la recherche médicale. Mais plusieurs cas bien documentés révèlent que la soif du profit passe bien aux devants des raisons humanitaires. C'est le cas ici au Québec de plusieurs médicaments non remboursés par la RAMQ, et dont les prix dépassent l'entendement.  Avec Cybèle Olivier et Charles Trahan  Pour de l'information concernant l'utilisation de vos données personnelles - https://omnystudio.com/policies/listener/fr

Entrevue avec Alexandre Chagnon, pharmacien.  Une petite fille du Saguenay a bénéficié du médicament le plus cher au monde, le Zolgensma, pour traiter son amyotrophie spinale. Quel est ce médicament et pourquoi est-il si onéreux? Pour de l'information concernant l'utilisation de vos données personnelles - https://omnystudio.com/policies/listener/fr

Entrevue avec Me Paul Brunet, président-directeur général du Conseil protection des malades.  Marguerite Blais a annoncé hier que les aînés hébergés en RPA ou en CHSLD pourront profiter d'allègements concernant le nombre de visiteurs autorisés et le maximum de personnes permises dans les salles à manger. Au menu de la rencontre entre Marie-Claude Barrette et Sophie Durocher, on craint que le rapport de la Commission Laurent ne se retrouve tabletté alors que rien ne bouge et que le bien-être de l'enfant n'est toujours pas au centre de la prise de décision le concernant.  Entrevue avec Fabien Major, Planificateur financier et conseiller en gestion de patrimoine chez Gestion de capital Assante - Équipe Major.  À l'approche de la date limite pour cotiser à un REER pour l'année d'imposition 2021, Fabien Major répond à nos questions de finances personnelles. Quelle est la différence entre un REER et un CELI? De combien d'argent a-t-on besoin pour une retraite confortable?  Entrevue avec Alexandre Chagnon, pharmacien.  Une petite fille du Saguenay a bénéficié du médicament le plus cher au monde, le Zolgensma, pour traiter son amyotrophie spinale. Quel est ce médicament et pourquoi est-il si onéreux? Pour de l'information concernant l'utilisation de vos données personnelles - https://omnystudio.com/policies/listener/fr

FOLGE 73 - "Frikadellen Asystolie" Grüße von der Intensivstation, der Arztpraxis und dem Marketingbüro! Wie haben Moritz und Kamil sich eigentlich kennengelernt? Das erfahrt ihr im Lagerfeuertalk. Ausserdem: Moritz ist diese Woche am emotionalen Down seines Lebens angelangt und jetzt kommt auch noch Karneval! Wir sprechen bei unseren "Awesome 3" über die seltensten Erkrankungen und Syndrome der Welt und über unsere eigenen Erfahrungen zu dem Thema. Und zu guter Letzt: Kennt ihr die teuerste Spritze der Welt? Wir schnacken über Helfersyndrome, Hypochondrie und Vorurteile in der Klinik. Kennt ihr das "Mamma Mia Syndrom" ? ... Let's talk about it! Welche Geräusche kann Kamil mit seiner Misophonie absolut nicht ertragen? Themen über Themen, Schnack über Schnack. Genießt Euren Sonntag mit unserer neuen Folge!

SMA News Today's multimedia associate, Price Wooldridge, reads an article about how a small group of children given the gene therapy after Spinraza's start showed only slight further gains; early treatment most important. Also, in her latest vlog DeAnn Runge explores the topic of furry friends. Pets are an important aspect of her life. She explains how she feels pets can benefit the lives of anyone living with SMA. Watch here: https://youtu.be/9Etax9E4cH0 Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/.

Zolgensma is a life-saving drug but it costs more than a million and a half pounds per patient. Who decides if the NHS can afford it? See acast.com/privacy for privacy and opt-out information.

The Circle of Kindness Foundation, which helps children with rare illnesses, was created in early 2021 at Vladimir Putin's own initiative. It's funded by a two percent tax on the income of Russia's wealthiest citizens. The organization pays for expensive medications -- but competition is rare at the auctions where these drugs are procured. One of the foundation's main suppliers is a company called Irvin -- and it's connected to both the foundation's head and to the family of Alexey Dyumin, Putin's former bodyguard. In addition, the husband of the woman responsible for purchasing medications on behalf of the Circle of Kindness works for the owner of the company that supplies the foundation with Zolgensma -- the most expensive drug in the world. Meduza correspondents Svetlana Reiter and Maria Zholobova break down how the Circle of Kindness Foundation's procurement system works and why its board of trustees (which includes well-known actors and philanthropists) believes it lacks transparency. Original Article: https://meduza.io/en/feature/2021/12/30/a-very-close-knit-circle

SMA News Today's multimedia associate, Price Wooldridge, discusses how Novartis Pharmaceuticals Canada applauds the province of Quebec for its move to offer public reimbursement for the SMA gene therapy Zolgensma. Also, making meaningful connections has become difficult for DeAnn Runge over the past several years. She explains why that is and shares some of her recent experiences. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

Cuma sabahından günaydın! Dünya'dan ipuçları ile başlıyoruz;-Rusya, SMA hastalarının tedavisi için İsviçre'de geliştirilen Zolgensma ilacını tescil etti.-Suudi Arabistan ve Katar, iki ülkenin güvenliğini ve istikrarını korumak için anlaştı.-Sudan‘da kaçırılan iki Türk vatandaşı kurtarıldı.-AB Komisyonu, 15 AB üyesi ülkenin toplam 40 bin Afgan'ı kabul edeceğini duyurdu.-Putin: Ukrayna'nın Donbass bölgesinde yaşananlar soykırımı andırıyor.-AB ülkeleri, Hırvatistan'ın Schengen bölgesine katılmasına onay verdi.-Nebati: Herkesin beklentilerini karşılayacak bir artış yapmayı planlıyoruz, işçi kardeşlerimiz rahat etsin.-Forbes'un dünyanın en güçlü kadınları listede bilim insanı Özlem Türeci ve iş insanı Güler Sabancı yer aldı.

SMA News Today's multimedia associate, Price Wooldridge, discusses how the U.S. FDA has added acute liver failure to the list of concerns with use of Zolgensma in children with spinal muscular atrophy. Plus, the value of time can be a difficult concept to master. In Ari Anderson's recent article, “Time Is a Balancing Act, Let's Learn How to Do It Together,” he talks about the value of time and how he prioritizes his precious time. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

The Russian Health Ministry has registered the world's most expensive drug -- a multi-million dollar gene therapy medication, sold under the brand name Zolgensma. Original Article: https://meduza.io/en/news/2021/12/09/russia-registers-multi-million-dollar-gene-therapy-drug-zolgensma

Post-marketing requirements within the European submission process offer conditional approval more flexible than FDA. Rachel Smith, Portfolio Director for Veristat, discusses the three types of post-marketing requirements that can be requested by the EMA and draws upon the recently approved therapies – Tecartus, Zolgensma and Libmeldy, Skysona and Abecma– for real-world context. 

SMA News Today's multimedia associate, Price Wooldridge, discusses an article about how a Canadian agency found evidence lacking to support gene therapy reimbursement in older babies and toddlers with spinal muscular atrophy. Plus, DeAnn Runge talks about how sometimes being inundated with all things SMA gets to be too much for her. Today she's talking about a topic totally unrelated to SMA to take her mind off it for a brief moment. She's looking for creative input for a project she has in the works. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

Hablamos con el padre de Bauti, un bebé que padece AME 1 y necesita uno de los medicamento más caros del mundo llamado "Zolgensma".

SMA News Today's multimedia associate, Price Wooldridge, discusses how Spinal Muscular Atrophy (SMA) children not helped by Zolgensma, are being enrolled in the Spinraza RESPOND trial. Plus, usually Kevin Schaefer reads his own columns, but today DeAnn Runge has the opportunity to share his latest column. Titled, “Embracing a New Chapter in Life With SMA,” Kevin writes about his longtime caregiver leaving and all the emotions surrounding it, of course putting his unique spin on it. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

(Attention, propos difficiles) Comment expliquer à des parents qui découvrent que leur bébé, atteint d'une maladie rare, ne peut recevoir le traitement qui pourrait le sauver ? Comment leur expliquer que ce traitement à 2 millions d'euros n'est pas homologué ou pris en charge par les assurances ? Qu'ils devront s'en passer et voir leur enfant mourir à petit feu ? Ces quelques lignes sont extrêmement dérangeantes, perturbantes et scandaleuses à lire. Mais elles reflètent la triste réalité des parents de bébés atteints d'amyotrophie spinale musculaire. Cette maladie atteint leurs muscles et provoque une dégénérescence, les rendant faibles jusqu'à ce que leurs organes cessent de fonctionner et qu'ils meurent. Tout cela pourrait être évité si tous les bébés et enfants atteints de cette maladie pouvaient avoir accès au traitement existant actuellement : le Zolgensma. Malheureusement, entre les contraintes de poids – certains médecins ne l'administrent qu'en-dessous de 11kg, certains acceptent jusqu'à 21kg - et les contraintes financières – le coût du Zolgensma est de 2 millions d'euros non remboursés par les assurances – beaucoup d'enfants n'y ont pas accès et meurent finalement de cette maladie. C'est entre autres pour lutter contre cette injustice et pour aider à récolter des fonds et soutenir les familles de ces enfants qu'Aline et Joanna ont monté l'association SMA Warriors. Alors n'hésitez pas à les contacter pour savoir comment les aider car comme le dit Aline, chaque geste compte.  

SMA News Today's multimedia associate, Price Wooldridge, discusses the best possible outcome for infants with Spinal Muscular Atrophy (SMA) is newborn screening, then Zolgensma, according to a cost-effectiveness study in Australia Also, content creator DeAnn Runge talks about getting her Jaco Robotic arm. She shares her concerns and the adjustments she's had to make after receiving it. Despite that she says it's worth the effort. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

Learn how NFTs can be used for good! Marina Granger interviews Dina Brodsky and Rafael Hoekstra as they team up to fight against Spinal Muscular Atrophy by minting an NFT image of 1000 artworks - all images from Instagram posts using the hashtag #BirdsforSofiaNFT. All proceeds will go towards getting a dose of Zolgensma for a baby Russian orphan, Darina Sorokina. Tune in! Use the hashtag #BirdsforSofiaNFT when posting an artwork **of a bird** on Instagram and visit BirdsforSofia.org to find out how else you can help!

Description: Viagra, Crestor, Eliquis, and many other brand-name medications come to mind when the discussion turns to expensive drugs. These drugs are pocket change in comparison to Zolgensma, a new(ish) gene therapy for Spinal Muscular Atrophy that costs as much as a private island. Tune in as our hosts discuss this incredible drug and some of the ramifications of its incredible price tag. This is NOT your physician's podcast. Hosts Shane Garrettson and Cal Vandergrift dive into the pharmacy world with fun, interesting, and downright weird topics! Tune in for NEW episodes, available on Spotify, Apple, Anchor, and more! Check out our Facebook, Twitter, and Instagram pages at Let's Pharmonize to view videos and images relevant to every episode! If you have any questions, comments, or even corrections, e-mail us at pharmonization@gmail.com. PLEASE READ: Shane and Cal are NOT medical professionals. DO NOT USE the information presented in this podcast to aid in your own personal health or medicinal benefit. This is a light-hearted podcast that should not be taken with the same seriousness as your own personal health, A special thanks to Kelly Kerr for creating the music used in the intro and outro. Additional music by FesliyanStudios See omnystudio.com/listener for privacy information. Learn more about your ad choices. Visit megaphone.fm/adchoices

FirstWord Pharma PLUS editor Simon King discusses the approval of Bristol Myers Squibb and bluebird bio's CAR-T therapy Abecma for multiple myeloma with senior Therapy Trends analyst Sarah Harris, speaks to Novartis' European gene therapy chief Mike Fraser about the launch of Zolgensma and asks FirstWord HealthTech's Tina Tan why the US Federal Trade Commission is looking to block Illumina's proposed acquisition of Grail in the cancer diagnostics market.

The ability to target the underlying cause of a disease and make a lasting correction makes gene therapy an attractive approach to treating neurodegenerative conditions. The advent of Zolgensma, a gene therapy for the treatment of the rare neurodegenerative condition spinal muscular atrophy, serves as a model for this approach. A recent review article in Nature Neuroscience looks at the advances in development of gene therapies for neurodegenerative disease and considers the challenges and promises. We spoke to article co-author Subhojit Roy, professor in the departments of Pathology and Neuroscience at the University of California, San Diego, about the pace of activity in this area, why he believes it is so promising, and its potential extend beyond monogenic diseases. This podcast is part of our ongoing Platforms of Hope series that explore advances in gene Thanks to Pfizer, Inc., Bluebird, and Novartis Gene Therapies for their support of this podcast article, part of our Platforms of Hope: Advances in Gene Therapy and Gene Editing series.therapy and gene editing. Thanks to Pfizer, Inc., Bluebird, and Novartis Gene Therapies for their support of this podcast, part of our Platforms of Hope: Advances in Gene Therapy and Gene Editing series.

One disease. Two families. Several different outcomes. Victoria Strong and Amy Medina share their experiences with Spinal Muscular Atrophy (SMA) Type 1 and how putting hope into action can change a disease forever.Links from today's episode:Read more of Victoria's story, learn about the Gwendolyn Strong Foundation, or shop Victoria's clothing brand at www.nevergiveup.org. Learn more about Amy Medina and her family at their CaringBridge site.Learn more about SMA here.