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MedNess – The highs in cancer and sickle cell anemia treatments

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MedNess is back with big news from the pharma companies. CAR-T therapy is getting closer to the market and sickle cell anemia sees a new yet ‘not-so-new’ therapeutic development. Stock prices of Indian pharma giant Biocon  soar to a record high with a recommendation for a biosimilar that can be used for treating breast cancer. Do not miss out on the details of what these companies have in mind, and how are their competitors faring.

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MedNess North America

Novartis poised to win CAR-T race

In a historic move on July 12, the US Food and Drug Administration (FDA)’s Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of Novartis’ CTL019 (tisagenlecleucel-T), a groundbreaking CAR-T therapy for treating relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in children and young adults. This decision is expected to lead to approval of the therapy by US FDA on or before October 3.

Due to the complexity of the therapy and expert care needed to manage side-effects, Novartis upon approval will restrict CTL019 use to 30-35 treatment centers in the U.S. who will be trained on a comprehensive risk-mitigation strategy that drew high praise from several ODAC members during the hearing. Novartis has plans for additional filings for CTL019 later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL).

Close rival Kite Pharma’s CAR-T application for the treatment of adults with advanced and aggressive lymphoma will receive a decision from FDA in November. Other key players being keenly watched in this field are Juno Therapeutics, Bluebird Bio, and Cellectis.

The announcement, however, did not affect Novartis’ or Oxford BioMedica’s shares, the companies supplying raw materials for CTL019. Before this announcement, Oxford BioMedica renewed its contract with Novartis extending its partnership for the commercial and clinical supply of its LentiVector gene delivery technology. On the other hand, Kite Pharma shares have seen big swings and is currently trading 24% higher than Novartis.


FDA approves first sickle cell disease treatment in 20 years


On July 7, the US FDA approved Endari for patients with Sickle Cell Disease (SCD) to reduce severe complications associated with the disorder. Endari, developed by Emmaus Lifesciences Inc. based in Torrance, CA, is the first FDA-approved treatment for pediatric patients with SCD and the first new treatment in almost 20 years for adult patients. The therapy is an orally administered pharmaceutical grade L-glutamine. The therapy’s efficacy and safety were evaluated in a 48-week Phase 3 trial (NCT01179217) involving sickle cell patients between the ages of 5 and 58 who had two or more painful crises in 12 months prior to enrollment. Results from the trial demonstrated that Endari reduced the frequency of sickle cell crises by 25 percent and hospitalizations by 33 percent.

Sickle cell disease is a rare, inherited disorder characterized by abnormally sickle-shaped red blood cells. These malformed red blood cells clog blood vessels and cut off oxygen to the body’s tissues, leading to episodes of severe pain and organ damage. Approximately 100,000 people in the U.S. have sickle cell disease, according to the National Institutes of Health. Endari helps increasing free glutamine in blood, which is taken up by the sickle red blood cells and used to generate anti-oxidant molecules as a product of glutamine degradation. These new antioxidants help neutralize the oxidative stress in sickle cells, allowing them to regain the flexibility needed to travel through blood vessels and capillaries, carrying oxygen to tissues.
L-glutamine, the active ingredient in Endari can be purchased over the counter, which could complicate Emmaus’s ability to obtain insurance coverage. Ontario-based Generex Biotechnology acquired controlling interest in Emmaus LifeSciences in March 2017. The recent FDA approval saw an 8% increase in share value for Generex Biotechnology.

MedNess Asia

Biocon flying high with US FDA recommendation



Biocon’s biosimilar of Trastuzumab, a breast cancer drug obtained unanimous recommendation (16-0) from US FDA ODAC – taking the drug closer to procuring US FDA’s marketing approval. Trastuzumab, originally developed by Roche is commonly used to treat HER2-positive breast cancer and has a multi billion-dollar market globally with an estimated market of $6.9 bn in the US alone. Biocon chairperson Kiran Mazumdar-Shaw said that they expect to obtain approval to launch their product by September 3.

Biocon collaborated with US drug maker Mylan to develop this biosimilar. Biocon will manufacture the product for all markets globally with a profit sharing arrangement with Mylan for its markets. However, with numerous observations from US FDA and European regulators, Biocon faces concerns regarding quality lapses. To circumvent these Ms. Mazumdar-Shaw mentioned that the company has GMP certification for its drug substance facility for trastuzumab and will expedite the process to seek an early re-inspection.

It is important that the approvals work swiftly for Biocon due to the risk of losing market share to other generics manufacturing competitors . One company to watch out for will be Aurobindo Pharma – the newest entrant in oncology segment in India. It plans to file for 15-17 products in oncology this year and expects to be able to start selling by end of the year. Last month, Dr. Reddy’s Laboratories and its partner Natco Pharma obtained US FDA approval for the generic version of Doxil, a chemotherapy drug used for ovarian cancer, Kaposi’s sarcoma, etc. to launch in the US market.


While the US FDA approval recommendation has already caused a surge of 11.8% in the stock prices of Biocon, it will need to concentrate highly on strengthening its technical capabilities and avoid quality lapses. It cannot afford to lose time in getting the approvals in order to avoid losing the first mover’s advantage in the market. Aurobindo Pharma has already seen a surge of 22.8% since it announced plans to file its oncology products.









About the authors:

Radhika Gopal is currently a postdoctoral research associate at The Scripps Research Institute, La Jolla, where she works on Hepatitis C virus glycoproteins. She also chairs the Corporate Sponsorship Committee for the San Diego chapter of AWIS (Association for Women in Science). Radhika has completed project management and business fundamentals courses from UCSD, and is actively seeking opportunities in the Biotech Industry. In her spare time, she enjoys music, hiking, reading music and the beach. She can be reached here.

Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

About the editor:

Sayantan Chakraborty is an IRTA postdoctoral visiting fellow at the National Institute on Aging – National Institutes of Health, Baltimore, USA. Apart from science, he invests his time in networking, writing, and organizing events. He is consolidating his efforts to build a platform that brings together scientists and industry professionals and to help spread the perception of alternate careers for life science graduates.

Follow him on Twitter @ch_sayantan


© The contents of Medness are the copyright of the PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs)

Featured image: Ipsa Jain (you can find more of her works on Facebook and Instagram)

Spread of evil – Cancer cells in blood stream by IpsaWonders

Blog design: Abhinav Dey

Creative Commons License
This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

MedNess Special: Bringing North America, Europe and Asia together

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Hello everyone and welcome to MedNess Global! Here at ClubSciWri, we are constantly trying to present the most impactful news in an ever-evolving innovative fashion. In this special edition, we bring business news from pharma and healthcare sectors in North America, Europe and Asia. Know what defines the industry space in each continent. Where is big capital invested on pharma research? And where does the manufacturing happen? What regulations underlie the drug making and selling processes across continents? This is an effort to give our readers a holistic understanding of international collaborations and deals in the industry. We thank our additional team of writers for their input; MedNess North America: Vinita Bharat; MedNess Europe: Czuee Morey; and MedNess Asia: Somdatta Karak


We value your input, please do not forget to provide your feedback (Fill the form at the end of this blog). Please subscribe to ClubSciWri ( for weekly updates!

MedNess North America

Merck Ventures supported Inthera Bioscience with their $11M investment

Protein-protein interactions (PPIs) are the basis of multiple control points in the body and play a pivotal role in multiple diseases, including cancer. PPI’s serve as the important target classes in oncology but are extremely intractable to work with.  Inthera bioscience, a Swiss based biopharma aims to design oral small molecules which can inhibit these PPIs, leading to the targeted approach for the treatment of solid tumors.  

For this innovative, novel and promising approach, Inthera drew the seed money of $3.8M in 2005, and now once again Merck Ventures invested $10.8M to wrap up pre-clinical developments focusing on HPV-associated cancers and hypoxia-inducible signaling  (FierceBiotech).

MedNess: Inthera Biosciences is a private biopharmaceutical company focussing on PPI’s. Inthera’s research group focuses on HPV-associated cancers and hypoxic signaling. Merck Ventures is the venture capital wing of Merck. This wing invests in novel products and technologies with significant potential to impact Merck’s core research areas (Biospace)

Novartis fully concentrated on its CAR-T cell therapy for “ALL” cancer

Novartis is specifically betting on its CAR-T cell therapy even though acknowledging that there is a gap in the other major I-O driven therapies like, IDO, BTK and PARP inhibitors. CAR-T is patient specific and looks likely to be the first of this new cancer class to reach the market by this fall.

CAR-T cell therapy involves the drawing of T-cells from patient’s blood and then reprogramming it in laboratory to hunt patient’s cancer cells and other B-cells expressing a particular antigen. Novartis touts this week data of its CAR-T candidate, CTL119 which when used with Janssen/Pharmacyclics’ Imbruvica (ibrutinib) gave complete responses with no evidence of disease in the bone marrow at three months (FierceBiotech).

MedNess: Novartis on June 1 opened with 81.78 and closed by 81.86 ( Novartis stock price has increased by 12.3% in the last 12 months. According to Zack’s classified, Novartis ranks number 3 and with its innovative technology, the stocks are advised to be hold onto (;


Tesaro’s new FDA approval med Zejula is waiting for its response

Tesaro’s PARP inhibitor based drug Zejula has one of 2017’s biggest launches, with an estimate of $1.9 billion till 2022. The company requested offers from the potential bidders but seems like they are waiting to hear from them.

Apart from the high price of this drug, buyers seem to wait for ASCO meeting where they can compare the results of Zejula with AstraZeneca’s Lynparza, Merck’s Keytruda and others. Hence, Zejula clealry opens up a PARP market and its big response is now relying on its ability to rack up new indications in prostate and breast cancer (FiercePharma).

 MedNess Europe

Therapeutic antibody against non-Hodgkin’s lymphoma developed by MorphoSys enters Phase 3 clinical trials

The German biotech MorphoSys announced that its therapeutic antibody, MOR208, has entered Phase 3 clinical trials after successful completion of the phase 2 part of the combined phase 2/3 trials. The randomized, multicenter phase 2/3 study is designed to investigate the efficacy of MOR208 plus bendamustine versus rituximab plus bendamustine in patients with relapsed or refractory diffuse large B cell lymphoma (R/R DLBCL), the most common form of non-Hodgkin’s lymphoma, who are not eligible for high-dose chemotherapy and autologous stem cell transplantation.

MOR208 is an Fc-enhanced monoclonal antibody that targets CD-19 homogeneously expressed on the surface of all B-cells. MOR208 is intended to induce direct apoptosis by binding to CD-19 along with significant potentiation of antibody-dependent cell-mediated cytotoxicity and cellular phagocytosis, thus aiming to improve a key mechanism of tumor cell killing.

MOR208 can therefore be useful to treat various B-cell malignancies such as non-Hodgkin’s lymphoma and chronic lymphocytic leukemia (CLL). Each year about 150,000 people are afflicted by B cell malignancies in the seven major markets. MOR208 was granted orphan drug designation in DLBCL and CLL across US and Europe. Additionally, the FDA has granted fast track designation for the treatment of DLBCL. MOR208 is under investigation in a separate clinical trial for CLL.

The dosing of the first patient in the phase 3 part will trigger an undisclosed milestone payment to Xencor, Inc., from whom MOR208 was in-licensed in 2010. MorphoSys has worldwide rights to MOR208. If this clinical trial is successful, MOR208 will be the first therapy that MorphoSys develops in clinical trials independently. However, MorphoSys has had a lot of success with its Human Combinatorial Antibody Library (HuCAL) that generates human antibodies in bacteria, after having been screened with the aid of specific phages. In partnership with Big pharma companies, MorphoSys has funneled over 100 monoclonal antibodies into the clinic with this platform (, Morphosys website).

MedNess Asia

Mismatch between FDA regulations and its implementation in Chinese API manufacturing companies

Cheaper manufacturing processes in China and India attract pharma companies in getting their Active Pharmaceutical Ingredients (APIs) manufactured for cheap in Asia. However, the manufacturing companies have to comply with regulations of the country where the drug is sold and intended to be used. Of the companies facing non-compliance with this are Chinese API makers, Teva Pharmaceutical Industries and Changzhou Jintan Qianyao Raw Material Factory failed US FDA inspection and has consequently been banned to ship all their products to the USA. The company had failed to meet with the Good Manufacturing Practice (GMP) expectations from  the US FDA. (GEN, FiercePharma)

MedNess: China is currently the world’s leading producer of pharma ingredients, covering 40% of global production, primarily because of the price advantage that they offer. It will be interesting to see if the API companies are willing to increase their costs by adding more regulatory expenses or let go of the multinational companies. On the other hand, China FDA (CFDA) has tightened its regulations to stop sub-standard operations.

Pricing pressures and non-compliance with US FDA regulations make difficult times for Sun Pharma

At the end of FY2016, Sun Pharmaceuticals Ltd., India’s largest pharma company, took a hit in its US sales for numerous reasons. Its key API maker for the US market, Halol faces US FDA regulatory limitations since 2014. US FDA slammed Halol with warning letters twice for reasons from inadequate data protection on computer to possibilities of contamination in their products despite Sun seeking help from consultants to make the plant US FDA compliant. In addition, Sun faces pricing pressure like the other generic drug companies. In addition, its US subsidiary specialty generics drugmaker, Taro faced a 26% drop in their sales.

More than 70% of Sun’s sales are from outside India, with US contributing to 50% of its turnover. With multiple worries continuing, Sun’s Managing Director Dilip Sanghvi has warned of single digit declines in the FY2018 revenues. (FiercePharma, Business Standard)

MedNess: Over the last 6 months Sun Pharma has seen a 28% drop in its stock prices (NSE). While resolving US FDA warning letters can take long, generic drugs pricing pressures might bring more mergers and acquisitions in the field.


China’s steps beyond generic drug making in healthcare sector

Founded in 2012, in Beijing, privately-held and venture-capital (VC) backed, CANbridge Life Sciences Ltd. has paved its path towards developing leading candidates in solid-tumor oncology, primarily in China, Taiwan and Korea by gathering funding worth $40mn, together after its second round of funding. Currently it focuses on two of its prime compounds – CAN008 and CAN017, and the former is already in phase I/II trial for glioblastoma multiforme, in Taiwan and will begin its phase II in China in 2018. CAN017 is designed against esophageal squamous cell cancer – the most prevalent form of cancer in Asia and has already been successful in phase I study in Greater China. CANbridge partners with AVEO Oncology (for the rights on the compounds) and Böhringer Ingelheim (for manufacturing) in development of CAN017. CANbridge talks of plans of more strategic transactions in 2017 – backed by strong finances and executive team – in direction of developing specialty healthcare products.  (Fierce Biotech, Biospace)

MedNess: China’s huge aging population and increase in lifestyle disorders as well as growth in per capita earning and expenditure is a promising ground to get into specialty healthcare products in China. CANbridge is an interesting example to study commercialization of healthcare products that are under-served in China and North Asia, by partnering with its developers and manufacturers in western countries. Since CANbridge is still VC backed, stocks are not yet for sale.



Featured Image by: Vinita Bharat








About the authors:







Imit Kaur, Ph.D. is a freelance scientific advisor, medical writer, editor, and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development. Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)







Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.

Czuee Morey has a PhD from University of Lausanne and pursued a postdoc from University of Geneva. She has broad interests in healthcare and technology as evidenced by her research experience in fields such as protein biophysics and genomics. She has previously worked as a patent analyst and also built the business plan for her startup project. She is currently working in market intelligence for digital healthcare at Debiopharm in Lausanne, Switzerland.

Vinita Bharat Ph.D., is currently a postdoctoral research fellow at European Neuroscience Institute, Göttingen, Germany and had been an International Max Planck Research School (IMPRS) student here. Her research area focuses on cellular and molecular neuroscience. Other than enjoying ‘being a scientist’, she has also been working on science education. Presenting science in easy and fun way is what she loves doing through her platform “Fuzzy Synapse” (one can find fuzzy synapse on Facebook, Instagram and Twitter). She is a fun, enthusiastic and curious person, passionate about traveling, loves celebrations and bringing smiles around her.

© The contents of Medness are the copyright of the PhD Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs)

Blog design: Abhinav Dey

Creative Commons License
This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

FDA Breakthrough A’La CAR-T: Medness Focus on Novartis CTL019

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What’s the big news?

Novartis announced that the US Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational Chimeric Antigen Receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

What’s even bigger?

On April 18 (2017) CTL019 received the FDA granted breakthrough therapy designation for the treatment of adults with relapsed and refractory diffuse large B-cell lymphoma who failed two or more prior therapies. Read more

What is ALL?

Image reference

Acute Lymphoblastic Leukemia (ALL)

Each part of its name tells you something about the cancer itself:

    • Acute: Often fast-growing, requires early detection and treatment. Without treatment, bone marrow cells developmentally impaired, resulting in an unhealthy bone marrow filled with proliferating abnormal lymphocytes.
    • Lymphoblastic: Affects the lymphocytes of a patient’s white blood cells. Alternative term is lymphocytic.
    • Leukemia: Leukemia is a cancer of the blood cells.
  • Most common cancer in children, but it can also occur in adults of all ages (bimodal age distribution, with peaks at 3-7 years and 65 years of age).
  • Clinical presentation is nonspecific:
    1. fever;
    2. infection;
    3. bleeding;
    4. bone pain;
    5. lymphadenopathy;
    6. CNS involvement.
  • Disease classification based on evaluation of cells derived from a bone marrow or tissue biopsy. Clonal cells may be B cells (B-precursor lineage, 75%) or T cells. There are three main different ALL subtypes as follows:
    1. Pre (precursor) B cell ALL – most common in adults
    2. Mature B cell ALL – identified by particular genetic changes
    3. Pre (precursor) T cell ALL – more likely in young adults and more common in men
  • Management involves
    • remission induction with combination chemotherapy.
    • intrathecal chemotherapy is indicated for all patients to prevent CNS relapse.
    • Post-remission, patients undergo 1-3 years of maintenance therapy to eliminate residual disease.
    • Read more


What’s the history ?

  • CAR T-cell therapy, may appear to be overnight success, has a long experimental history.  Chemist and immunologist, Zelig Eshhar, developed the first CAR-T cells at the Weizmann Institute of Science in Israel in the late 1980s. In 1990, Eshhar took a year-long sabbatical, joined Steven Rosenberg at the National Institutes of Health, and prepared CARs that targeted human melanoma. “We designed CAR T cells to overcome a number of problems in getting T cells to attack cancer,” says Eshhar. The problems being a tumor’s ability to escape immune recognition by preventing the major histocompatibility complex molecules and the immunosuppressive tumor microenvironment.

  • CTL019 first developed by the University of Pennsylvania (Penn) by Carl June‘s group (link to original NEJM paper). Read more.
  • In 2012, Novartis and Penn created a global collaboration to advance research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications. In March 2017, Novartis announced that the FDA accepted the company’s Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.


What is the science behind it?

  • CAR-T therapies exploit the capability of a patient’s immune system to fight their disease, (sometimes referred to as “fifth pillar” of cancer treatment).
  • Therapy involves engineering patients’ own immune cells to recognize and attack their tumors (popularly known as Adoptive Cell Transfer).
  • Renier J. Brentjens, MD (Memorial Sloan Kettering Cancer Center) describes it like “giving patients a living drug.”


What was the outcome of the clinical trials?

  • The Children’s Hospital of Philadelphia (CHOP) study (link) showed disappearance of all signs of cancer (a complete response) in 27 of the 30 patients treated. 19 out of 27 are still in remission
  • The NIH Pediatric Oncology Branch study (link) 14 of 20 patients had a complete response with 10 of them receiving successful stem cell transplant and remain cancer free.
  • The Memorial Sloan Kettering Cancer center (MSKCC) clinical trial study (link) 14 of the 16 participants showed complete response and 7 eligible patients got stem cell transplant staying cancer-free.
  • The NCI-led study (link) showed “Of 15 patients, eight achieved complete remissions (CRs), four achieved partial remissions, one had stable lymphoma, and two were not evaluable for response”. This showed the “effectiveness of treating chemotherapy-refractory B-cell malignancies with anti-CD19 CAR T cells”.
  • Novartis clinical trial (ELIANA) evaluating efficacy and safety of CTL019 (with study enrollment having occurred across 25 centers in the US, EU, Canada, Australia and Japan) found that 82% (41 of 50) of infused patients achieved complete remission.
  • The second global CAR-T trial, JULIET, following the Novartis ELIANA study, led FDA to confer Breakthrough Therapy Designation for Treatment of Adult Patients withrelapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL). The findings from JULIET are expected to be presented at an upcoming medical congress.

What are some of the doubts?

  • CAR-T, which induces an extreme immune response that attacks cancer cells, can create a cytokine storm leading to extreme side effects like high fever.
  • CAR-T might need the best – and presumably the most highly-paid – doctors and healthcare teams to ensure patients can manage the side effects.
  • The laboratory process of extracting immune system T-cells from each individual patient and altering the DNA to create chimeric antigen receptors will create additional costs (totaling upto $500,000-750,000 to treat one patient). Health providers might not be ready to foot the bill.
  • Initial failures from competitor Juno Therapeutics have created doubts on Novartis pulling out of the study. Novartis has previously backed out of large research programs like RNA interference.

What should the patients and their families know?

  • CTL019 is an investigational therapy- safety and efficacy profile not yet established.
  • Access to investigational therapies only available through carefully controlled and monitored clinical trials.
  • No guarantee that CTL019 will ever be commercially available anywhere in the world.

MedNess Quotient

After the announcement, Novartis shares were little changed but the shares of the company making CTL019 raw materials, Oxford BioMedica, rose by more than 4.5 percent. Alternatively, Kite Pharma, Novartis’ rival in CAR-T race, also submitted a rolling application for their chimeric antigen receptor T cell candidate. Rolling applications are allowed for promising new drugs. Kite’s application could be accepted early putting behind Novartis’. Therefore, the winner of the CAR-T race will set the price of the therapy and subsequently the stocks (Reuters and Nasdaq).

References and additional reading:


Featured image source: Twitter

Disclaimer: This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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