Thoracic Chemotherapy May Advance Mesothelioma Treatment
Thoracic surgeon Dr. Marcello Migliore is convinced that adding hyperthermic intrathoracic chemotherapy to aggressive surgery can extend survival significantly for patients with pleural mesothelioma cancer. He wants others to know that, too. This seldom-used, controversial procedure, also known as HITHOC, involves circulating a heated, high-concentration chemotherapy solution throughout the chest cavity for 60 minutes immediately following aggressive mesothelioma surgery. The idea is to kill any microscopic tumor cells that evaded the surgeon, hopefully slowing a potential cancer recurrence. Unlike HIPEC, a similar procedu...
Source: Asbestos and Mesothelioma News - January 19, 2021 Category: Environmental Health Authors: Fran Mannino Source Type: news

DARZALEX FASPRO ® (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis
January 15, 2021 (HORSHAM, Pa.) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1] DARZALEX FASPRO® is the first and only FDA-approved treatment for patients with this blood cell disorder that is associated with the production of an abnormal protein, which leads to th...
Source: Johnson and Johnson - January 16, 2021 Category: Pharmaceuticals Tags: Our Company Source Type: news

Research shows gene therapies can offer cost savings potential of up to $1.8M over five years for patients with certain blood diseases
Arecent analysis by Health Advances looks at two particularly burdensome hematological (or blood) diseases — beta thalassemia andhemophilia A— and finds that potential gene therapies in the pipeline could reduce per patient costs by as much as 41% and 90%, respectively, over five years. Research-based knowledge gained in recent years about the genetic foundation for these diseases has facilitated the pursuit of several promising gene t herapy approaches. (Source: The Catalyst)
Source: The Catalyst - January 15, 2021 Category: Pharmaceuticals Authors: Katie Koziara Tags: Rare Diseases Drug Cost New Era of Medicine The Value Collaborative Source Type: news

Intelligence deficit: Conclusion from the mouse to the human being
(University of Bonn) Impaired intelligence, movement disorders and developmental delays are typical for a group of rare diseases that belong to GPI anchor deficiencies. Researchers now used genetic engineering methods to create a mouse that mimics these patients very well. Studies in this animal model suggest that in GPI anchor deficiencies, a gene mutation impairs the transmission of stimuli at the synapses in the brain. The results are published in the journal PNAS. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 7, 2021 Category: International Medicine & Public Health Source Type: news

Redhill biopharma's RHB-204 granted FDA fast track designation for NTM disease
(RedHill Biopharma) RedHill Biopharma Ltd. (Nasdaq: RDHL), a specialty biopharmaceutical company, today announced that RHB-204 has been granted Fast Track designation by the US Food and Drug Administration (FDA) for its development as a potential first-line, stand-alone, oral treatment of pulmonary nontuberculous mycobacteria (NTM) disease caused by Mycobacterium avium Complex (MAC) -- a rare disease for which there is no FDA-approved first-line therapy. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 6, 2021 Category: Biology Source Type: news

How an ultra-rare disease is bringing this N.S. family closer together
Three members of a family living with the same rare disease say their shared experience has brought them closer together. Brad, Bradley and Amanda Bright are believed to be the only Canadians with Camurati-Engelmann disease. (Source: CBC | Health)
Source: CBC | Health - January 3, 2021 Category: Consumer Health News Tags: News/Canada/Nova Scotia Source Type: news

Centene completes acquisition of PANTHERx
Centene Corp. has completed a deal for national specialty pharmacy PANTHERx, which specializes in treating rare diseases and is based in Pittsburgh. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - December 30, 2020 Category: Health Management Authors: Vince Brennan Source Type: news

Centene completes acquisition of PANTHERx
Centene Corp. has completed a deal for national specialty pharmacy PANTHERx, which specializes in treating rare diseases and is based in Pittsburgh. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 30, 2020 Category: Biotechnology Authors: Vince Brennan Source Type: news

Centene completes acquisition of national specialty pharmacy
Clayton-based Centene Corp. has completed a deal for national specialty pharmacy PANTHERx, which specializes in treating rare diseases and is based in Pittsburgh. Terms of the deal were not disclosed, but Centene President and CEO Michael Neidorff said the deal will bolster his company’s portfolio, according to a release. "We are pleased to welcome the PANTHERx team to the Centene family," Neidorff said. "PANTHERx is a leader in rare disease pharmacy and brings a unique capability to our comprehensive… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - December 30, 2020 Category: Health Management Authors: Vince Brennan Source Type: news

Centene completes acquisition of national specialty pharmacy
Clayton-based Centene Corp. has completed a deal for national specialty pharmacy PANTHERx, which specializes in treating rare diseases and is based in Pittsburgh. Terms of the deal were not disclosed, but Centene President and CEO Michael Neidorff said the deal will bolster his company’s portfolio, according to a release. "We are pleased to welcome the PANTHERx team to the Centene family," Neidorff said. "PANTHERx is a leader in rare disease pharmacy and brings a unique capability to our comprehensive… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 30, 2020 Category: Biotechnology Authors: Vince Brennan Source Type: news

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease
Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy Final analysis confirmed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in pat... Biopharmaceuticals Protalix BioTherapeutics, Chiesi , Fabry disease, pegunigalsidase alfa (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 30, 2020 Category: Pharmaceuticals Source Type: news

FDA Approves Kineret (anakinra) for the Treatment of Deficiency of IL-1 Receptor Antagonist (DIRA)
WALTHAM, Mass., Dec. 22, 2020 (GLOBE NEWSWIRE) -- Sobi™, an international biopharmaceutical company dedicated to rare diseases, announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 22, 2020 Category: Drugs & Pharmacology Source Type: news

AstraZeneca shares fall on investor concern over $39bn deal
Some shareholders support acquisition of rare disease specialist Alexion (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - December 14, 2020 Category: Pharmaceuticals Source Type: news

AstraZeneca’s $39bn Alexion deal marks shift from prey to predator
Purchase of US rare disease specialist comes 6 years after pharma group saw off Pfizer takeover bid (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - December 14, 2020 Category: Pharmaceuticals Source Type: news

Covid-19 vaccine developer AstraZeneca agrees to buy rare disease firm Alexion for $39 billion
Leading Covid-19 vaccine developer AstraZeneca agreed on Saturday to buy Boston pharmaceutical firm Alexion for $39 billion. (Source: Reuters: Health)
Source: Reuters: Health - December 12, 2020 Category: Consumer Health News Source Type: news

AstraZeneca to buy Alexion in $39bn immunology deal
UK drugmaker to acquire US biotech specialising in rare disease treatment (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - December 12, 2020 Category: Pharmaceuticals Source Type: news

Study reveals distinct genomic landscape for young adults with appendiceal cancer
(Vanderbilt University Medical Center) The first study to compare molecular landscapes of early-onset and late-onset appendiceal cancer has revealed distinct non-silent mutations in the tumors of younger patients, setting the stage for the development of potential therapeutic advances for this rare disease. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - December 9, 2020 Category: Cancer & Oncology Source Type: news

Trench fever, rare disease that afflicted WWI soldiers, detected in homeless Winnipeggers
An infectious diseases specialist is calling on doctors to be aware of the signs of a rare illness he recently diagnosed four times among people experiencing homelessness in Winnipeg — one that commonly afflicted soldiers during the First World War. (Source: CBC | Health)
Source: CBC | Health - December 7, 2020 Category: Consumer Health News Tags: News/Canada/Manitoba Source Type: news

Rare Diseases Program
The Rare Diseases Program facilitates, supports and accelerates the development of drug and biologic products for the benefit of patients with rare disorders (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 1, 2020 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Extension of PDUFA Date for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease
CARMIEL, Israel, Nov. 27, 2020 -- (Healthcare Sales & Marketing Network) -- Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeuti... Biopharmaceuticals, FDA Protalix BioTherapeutics, Fabry disease, pegunigalsidase alfa (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 27, 2020 Category: Pharmaceuticals Source Type: news

Hack for NF winning projects announced
(Children's Tumor Foundation) Hack for NF, a hackathon for neurofibromatosis research hosted by the Children's Tumor Foundation, today announced the winners of the 2020 virtual event that brought together healthcare startups, developers, solutions architects, and hackathon enthusiasts for six weeks to drive scientific and medical innovation and improve the lives of patients living with neurofibromatosis and other rare diseases. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 24, 2020 Category: International Medicine & Public Health Source Type: news

Approval of pricey rare disease drug by Peninsula company has side benefit: Voucher it sells for $95 million
The drug, designed to change the course of a disease that typically kills kids by their mid-teens, will carry a net price will be about $650,000 a year. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 23, 2020 Category: American Health Authors: Ron Leuty Source Type: news

1st drug for rare rapid-aging disease extends kids' lives
The first treatment has been approved for a rare disease that causes rapid aging in children (Source: ABC News: Health)
Source: ABC News: Health - November 21, 2020 Category: Consumer Health News Tags: Health Source Type: news

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies
PALO ALTO, Calif., Nov. 20, 2020 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. Food and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - November 20, 2020 Category: Drugs & Pharmacology Source Type: news

Drug giant GSK spins out S.F.-based venture unit with $500M fund
The San Francisco-based venture fund of drug giant GlaxoSmithKline plc is stepping out on its own. SR One Capital Management is formally spinning out from GlaxoSmithKline (NYSE: GSK) with an oversubscribed $500 million fund targeting rare diseases to large diseases, cancer, immunology and neurodegenerative diseases. Those investments could include single-asset companies through platform technol ogies, said CEO Simeon George. The group, founded and funded solely by a GSK in 1985 as an autonomous… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - November 19, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

New tool that integrates the psychological, social and medical data of patients with rare diseases
(Universitat Oberta de Catalunya (UOC)) Researchers from the Universitat Oberta de Catalunya (UOC) and the technology center Eurecat have developed an innovative formal representation of rare disease data, including information unavailable in current models on the rare disease patient's biological, psychological and social profile. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - November 18, 2020 Category: Biology Source Type: news

Deadly Ebola-like Chapare virus CAN spread between people, CDC finds  
Known as the Chapare virus, the infection causes high fevers, abdominal pain, bleeding gums, eye pain and skin rashes. For the first time, the CDC found the rare disease spreads between humans. (Source: the Mail online | Health)
Source: the Mail online | Health - November 18, 2020 Category: Consumer Health News Source Type: news

Non-hereditary mutation acts as natural gene therapy in patient with rare disease
(Funda ç ã o de Amparo à Pesquisa do Estado de S ã o Paulo) Scientists at a research center supported by FAPESP identified a non-inherited mutation in blood cells from a patient with GATA2 deficiency that may have prevented bone marrow failure and other clinical manifestations. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - November 17, 2020 Category: International Medicine & Public Health Source Type: news

Africa: New, Rare Syndrome - With Covid-19 Seemingly a Precursor - Puts Children At Risk
[UCT] Multi-system inflammatory syndrome in children (MIS-C), a rare disease which seems to have COVID-19 as a precursor, has been found to develop in children a few weeks after the onset of the viral infection. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - November 13, 2020 Category: African Health Source Type: news

Rare disease drug maker plans to hire 100-150 at new gene therapy facility near Boston
Potential one-shot-and-cured gene therapy programs are increasing in clinical trials, putting pressure on third-party contract manufacturing organizations and the companies that use them. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 10, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

Janssen Headlines American Society of Hematology Annual Meeting With More Than 35 Presentations Highlighting Deep, Diverse Oncology Pipeline and Portfolio
RARITAN, N.J., November 5, 2020 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today more than 35 company-sponsored studies, including 10 oral presentations, will be featured at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition taking place virtually December 5-8, 2020.In multiple myeloma, highlights include Phase 1b/2 results for the B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) therapy ciltacabtagene autoleucel (cilta-cel); new and updated data for the anti-CD38 monoclonal antibody DARZALEX® (daratumumab) and the subcutane...
Source: Johnson and Johnson - November 5, 2020 Category: Pharmaceuticals Tags: Innovation Source Type: news

Making The Impossible Possible: A Conversation With Martine Rothblatt
Martine Rothblatt, creator of SiriusXM and whose company, United Therapeutics, has already saved thousands of lives from a rare disease, is working feverishly to create artificial organs, such as hearts, that could spare the lives of millions of people who can ’t get organ transplants. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - October 30, 2020 Category: Pharmaceuticals Authors: Steve Forbes, Forbes Staff Tags: Policy /policy Business /business Innovation /innovation Healthcare /healthcare Editors' Pick editors-pick Source Type: news

Citrine Medicine Advances Efforts to Create the Very First Rare Disease Ecosystem in China Through Strategic Partnership with Bioprojet to License Exclusive Rights for the Use of Orphan Narcolepsy Therapy Wakix(R) (pitolisant)
Citrine was founded by Eight Roads, F-Prime Capital, and Vivo Capital; and closed an $80 Million Series A financing earlier this year Company is focused on bringing important rare disease therapies to China while accelerating the rare disease drug devel... Biopharmaceuticals, Licensing Citrine Medicine, Bioprojet, Wakix, pitolisant, narcolepsy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - October 28, 2020 Category: Pharmaceuticals Source Type: news

Digital companions: helping improve patient treatment journeys
Early analysis of the unprecedented shift from traditional face-to-face healthcare towards virtual digital care during the pandemic show that patients have embraced this new care paradigm and expect it to endure beyond 2020.  Patient surveys carried out globally by therapeutics company, Medisafe, during March and April of this year revealed that more than 40% of patients turned to digital health for the first time during lockdown. According to data from Accenture, nine out of ten patients reported that the quality of their virtual care was as good or better than before.  Digital tools, such as video conferencin...
Source: EyeForPharma - October 23, 2020 Category: Pharmaceuticals Authors: Katie Osborne Source Type: news

Researchers uncover crucial gene for growth of Ewing sarcoma
(Center for Genomic Regulation) Researchers have discovered a gene that is critical for the development of Ewing sarcoma, a rare type of developmental cancer that presents in bones and soft tissues. Exploring the pharmacological inhibition of RING1B as a clinical therapy to treat Ewing sarcoma could open the door for new treatments for the rare disease. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - October 23, 2020 Category: Cancer & Oncology Source Type: news

Rare disease cures accelerator-data and analytics platform milestones highlighted
(Critical Path Institute (C-Path)) C-Path and NORD ® hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative agreement through the U.S. Food and Drug Administration (FDA), serves as a centralized and standardized infrastructure to support data sharing and data analysis to aid in the understanding of rare diseases. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 21, 2020 Category: International Medicine & Public Health Source Type: news

Patient engagement: Breaking through in digital
  Face-to-face engagement restrictions during the pandemic made remote engagement the only game in town for pharma and healthcare providers.  The speed at which both have risen to the challenge and adopted digital tools to solve urgent patient care and support needs has been striking. Most pharma players will have relaxed pre-pandemic standard operating procedures to get drugs and treatment advice to patients by any means.  And in a new Reuters Events Pharma survey, almost two thirds (63.2%) of respondents say their patient support programmes are able to meet current remote access conditions.  Now t...
Source: EyeForPharma - October 14, 2020 Category: Pharmaceuticals Authors: Andrew Stone Source Type: news

Replacing an enzyme to control a very rare disease
Until recently, there was no treatment specific to alpha-mannosidosis, one of the many rare diseases that jointly affect some 30 million citizens in Europe alone. Today, there is as EU-funded research developed enzyme-replacement therapy to stop the illness in its tracks, and this medicine is on the market. (Source: EUROPA - Research Information Centre)
Source: EUROPA - Research Information Centre - October 12, 2020 Category: Research Source Type: news

CDER Chief Reflects on Advances in Rare Diseases CDER Chief Reflects on Advances in Rare Diseases
In this interview, she reflects on the CDER's accomplishments in the field of rare diseases and what it's like to be overseeing the therapeutics component of Operation Warp Speed amid the COVID-19 pandemic.Medscape Medical News (Source: Medscape Infectious Diseases Headlines)
Source: Medscape Infectious Diseases Headlines - October 9, 2020 Category: Infectious Diseases Tags: Infectious Diseases News Source Type: news

Dr. Hahn's Remarks to the 2020 NORD Rare Disease Summit - 10/08/2020
Remarks by Stephen Hahn, MD Commissioner of Food and Drugs Dr. Hahn's Remarks to the 2020 NORD Rare Disease Summit Washington, DC Oct. 8, 2020 (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - October 9, 2020 Category: Drugs & Pharmacology Authors: FDA Source Type: news

With new biotech IPOs on the way, here's a look at the Bay Area companies that filed so far this year
From blank checks to already-acquired companies, and from cancer to rare diseases, the Bay Area life sciences IPO class of 2020 is largely trading above their initial offering prices. Can you see what they have in common? (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - October 8, 2020 Category: Health Management Authors: Ron Leuty Source Type: news

With new biotech IPOs on the way, here's a look at the Bay Area companies that filed so far this year
From blank checks to already-acquired companies, and from cancer to rare diseases, the Bay Area life sciences IPO class of 2020 is largely trading above their initial offering prices. Can you see what they have in common? (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 8, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

Big drug costs for small patients with rare diseases, study finds
(Michigan Medicine - University of Michigan) Only about one in every 170 children take them. But " orphan drugs " accounted for 1 in every 15 private insurance dollars spent on children's health care in the United States in 2018, according to a new study. That's up 65% from just five years before. And even though insurance companies pay much of the cost of high-priced orphan drugs that treat rare childhood diseases, families' share of the cost has risen rapidly. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - October 6, 2020 Category: International Medicine & Public Health Source Type: news

Center for Individualized Medicine ninth annual conference: Innovate. Connect. Transform.
It has been a remarkable year of discoveries and medical advances at Mayo Clinic's Center for Individualized Medicine. Some innovations include personalized treatments for cancer and rare diseases, and the development of lifesaving artificial intelligence algorithms. All of the center's efforts are driven by advanced genetic analysis and aimed at providing patients with answers, treatment [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - October 2, 2020 Category: Databases & Libraries Source Type: news

Up Close and Personal: A Life of Service - The Dr. Dirk Vander Mijnsbrugge Story
Sitting around the kitchen table, Dirk ’s family can’t help but talk about medicine. Dirk, his wife, all three of their children and their spouses are all doctors. With Dirk being the only one to go into industry, he was the target of much teasing. But not anymore. Here’s Dirk’s story.  The youngest son in a lower-middle class family, Dirk Vander Mijnsbrugge was raised to serve others. His parents modelled a life of service; his mom serving their family while his father served their country in the military.    Outside the family, his parents were deeply involved in social service an...
Source: EyeForPharma - October 2, 2020 Category: Pharmaceuticals Authors: Jill Donahue Source Type: news

Johnson & Johnson Completes Acquisition of Momenta Pharmaceuticals, Inc.
NEW BRUNSWICK, N.J., October 1, 2020 -- Johnson & Johnson (NYSE: JNJ) today announced it has successfully completed its acquisition of Momenta Pharmaceuticals, Inc. (“Momenta”), a company that discovers and develops novel therapies for immune-mediated diseases, in an all cash transaction for approximately $6.5 billion.“We’re delighted to welcome Momenta’s talented team to the Janssen Pharmaceutical Companies of Johnson & Johnson and to begin our work together to further advance patient care in autoantibody-driven diseases,” said Jennifer Taubert, Executive Vice President, Worldwi...
Source: Johnson and Johnson - October 1, 2020 Category: Pharmaceuticals Tags: Our Company Source Type: news

Rare Disease Drug Launch – Lessons Learned to Ensure Success is Not a...
In this free webinar, the featured speakers will discuss critical areas of focus for successful rare disease product launch and explain how to build strong connections with HCPs, patients, and...(PRWeb September 30, 2020)Read the full story at https://www.prweb.com/releases/rare_disease_drug_launch_lessons_learned_to_ensure_success_is_not_a_rare_event_upcoming_webinar_hosted_by_xtalks/prweb17432815.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 30, 2020 Category: Pharmaceuticals Source Type: news

Cincinnati Children's research helps pave way for newly approved use of drug
(Cincinnati Children's Hospital Medical Center) Following two decades of research on a group of rare diseases called hypereosinophilic syndrome at Cincinnati Children's Hospital Medical Center, the US Food and Drug Administration has approved the drug Nucala (mepolizumab) for use in the treatment of patients with hypereosinophilic syndrome. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 28, 2020 Category: International Medicine & Public Health Source Type: news

Blueprint Medicines Announces European Commission Approval of AYVAKYT(R) (avapritinib) for the Treatment of Adults with Unresectable or Metastatic PDGFRA D842V Mutant Gastrointestinal Stromal Tumors
CAMBRIDGE, Mass., Sept. 25, 2020 -- (Healthcare Sales & Marketing Network) -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that ... Biopharmaceuticals, Regulatory Blueprint Medicines, AYVAKIT, avapritinib, gastrointestinal stromal tumor (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - September 25, 2020 Category: Pharmaceuticals Source Type: news

Hugh Jackman Surprises 10-Year-Old And Bone Marrow Donor Who Saved Boy ’ s Life
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Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - September 25, 2020 Category: Consumer Health News Authors: Health – CBS Boston Tags: Boston News Health Syndicated CBSN Boston Syndicated Local Bone Marrow Donation Grady Smith Hugh Jackman Jessica Carroll. Source Type: news