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

GAVRETO (pralsetinib), FDA Approved for Treatment of Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer, Available at Biologics by McKesson
CARY, N.C., Sept. 17, 2020 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Blueprint Medicines as a specialty pharmacy provider for GAVRETOTM (pralsetinib) for the treatment of adult patients with metastatic RET fusion-p... (Source: McKesson News)
Source: McKesson News - September 17, 2020 Category: Information Technology Source Type: news

New Graphic: Public-Private collaboration fuels the U.S. biopharmaceutical ecosystem
America ’s biopharmaceutical companies are at the heart of a robust research and development (R&D) ecosystem that develops more innovative medicines than any other country in the world. In recent years, rapid advances in scientific discovery have ushered in a new era of medicine, transforming our ability to treat, and in some cases cure, some of the most challenging diseases, including cancer, rare diseases and autoimmune conditions. These advancements are due to the productivity of the United States ’biomedical R&D ecosystem, which is sustained by a policy framework that is designed to support and adva...
Source: The Catalyst - September 16, 2020 Category: Pharmaceuticals Authors: Jocelyn Ulrich Tags: Research and Development Intellectual Property & D Focus Source Type: news

From complexity to clarity in cell and gene therapy
One of the most exciting frontiers in medicine, cell and gene therapies are already offering breakthrough treatments and potential cures in severe genetic diseases and cancer. The innovations continue to advance rapidly, with press releases announcing major breakthroughs on a seemingly monthly basis.  It ’s undoubtedly a good news story but these advances bring with them the challenge of explaining all the exciting, but complex possibilities to patients and caregivers.  The groups that have historically been expected to help keep doctors up to speed cannot be expected to do so in this dynamic environm...
Source: EyeForPharma - September 16, 2020 Category: Pharmaceuticals Authors: Andrew Stone Source Type: news

Australia ’s field force: evolving on fast forward
The pandemic has, it will surprise no one, upended traditional ways of working for pharma ’s Australian field force. “I don't think that we will ever go back to working the way we did pre-COVID, ” says David Grolman, Medical Director at Pfizer Australia and New Zealand.”   But arguably this is less of a revolution than it might first seem. The havoc and disruption it has wreaked has merely accelerated change that was happening anyway, much of it positive, says Matthew Britland, VP of the Australian Pharmaceutical medical and scientific Professionals Association (APPA). “It’s c...
Source: EyeForPharma - September 16, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news

FDA grants rare disease designation to Clarity agent
Radiopharmaceutical firm Clarity Pharmaceuticals announced that the U.S. Food...Read more on AuntMinnie.comRelated Reading: Clarity's breast imaging agent enters clinical trial Clarity begins trial for pediatric neuroblastoma therapy Clarity, ImaginAb team to develop theranostic agents NorthStar inks supply deal with Clarity (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - September 10, 2020 Category: Radiology Source Type: news

New data show Roche ’s ENSPRYNG (satralizumab) significantly reduces severity and risk of relapse in neuromyelitis optica spectrum disorder (NMOSD)
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity ...
Source: Roche Media News - September 10, 2020 Category: Pharmaceuticals Source Type: news

New data show Roche ’s ENSPRYNG (satralizumab) significantly reduces severity and risk of relapse in neuromyelitis optica spectrum disorder (NMOSD)
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity ...
Source: Roche Investor Update - September 10, 2020 Category: Pharmaceuticals Source Type: news

Biopharma Leaders Unite To Stand With Science
NEW YORK, September 8, 2020 — The CEOs of AstraZeneca (LSE/STO/NYSE: AZN), BioNTech (NASDAQ: BNTX), GlaxoSmithKline plc (LSE/NYSE: GSK), Johnson & Johnson (NYSE: JNJ), Merck (NYSE: MRK), known as MSD outside the United States and Canada, Moderna, Inc. (Nasdaq: MRNA), Novavax, Inc. (Nasdaq: NVAX), Pfizer Inc. (NYSE: PFE), and Sanofi (NASDAQ: SNY), today announced a historic pledge, outlining a united commitment to uphold the integrity of the scientific process as they work towards potential global regulatory filings and approvals of the first COVID-19 vaccines. All nine CEOs signed the following pledge: We, the un...
Source: Johnson and Johnson - September 8, 2020 Category: Pharmaceuticals Tags: Our Company Source Type: news

Insights into treating rare cancer tumours benefit patients
What is the best way to tackle sarcomas? Although clinical trials help to generate insight, they are hard to set up for rare diseases. An EU-funded project has organised several trials focusing on these malignancies, providing insight that is already helping to save more lives. It has also paved the way for further research to help patients. (Source: EUROPA - Research Information Centre)
Source: EUROPA - Research Information Centre - September 7, 2020 Category: Research Source Type: news

Brain tumor drug receives rare disease designations from the FDA
(Oklahoma Medical Research Foundation) The Food and Drug Administration has given Orphan Disease Designation and Rare Pediatric Disease Designation to OKN-007, an experimental brain cancer treatment currently in Phase 2 clinical trials at eight U.S. sites. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - September 2, 2020 Category: Cancer & Oncology Source Type: news

NIDCR's Fall 2020 E-Newsletter
Having trouble viewing this email? View it as a Web page. NIDCR's Fall 2020 E-Newsletter In this issue: NIDCR News Funding Opportunities NIH/HHS News Funding Notices Science Advances Subscribe to NICDR News Grantee News   NIDCR News NIDCR Announces Availability of COVID-19 Research Funding On May 5, NIDCR issued two Notices of Special Interest highlighting the urgent need for research on coronavirus disease 2019. This research may be conducted either via the National Dental PBRN infrastructu...
Source: NIDCR Science News - September 1, 2020 Category: Dentistry Source Type: news

Dementia symptoms: Are signs developing at lightning speed? It could be a rare disease
DEMENTIA describes a group of progressive brain diseases that can, ultimately, become so debilitating that everyday life can never return to normal. A certain condition could propel the damage at mega speed. What is it? (Source: Daily Express - Health)
Source: Daily Express - Health - August 28, 2020 Category: Consumer Health News Source Type: news

FDA Approves Cystadrops (cysteamine) Ophthalmic Solution for the Ocular Manifestations of Cystinosis
LEBANON, N.J., Aug. 25, 2020 /PRNewswire/ -- Recordati Rare Diseases Inc., today announced the U.S. Food and Drug Administration (FDA) has approved Cystadrops (cysteamine ophthalmic solution) 0.37%. Cystadrops is a new, viscous eye drop solution... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 26, 2020 Category: Drugs & Pharmacology Source Type: news

Paying for cell and gene therapy - Is the future already here?
The ascent of cell and gene therapies over the past few years has been astonishing. And their rise looks unstoppable: By 2025, the FDA expects it will be reviewing 10 to 20 of these transformative drugs per year.    But as we listen to affordability concerns from payers, providers and patients, we ’ve also had to ask, perhaps a bit provocatively, how we can afford to pay for this boom in future cures?  " Without solutions to help payers manage the cost, some of our members may make the choice to exclude coverage " , stated insurer CVS Health earlier in the year in its position paper on ...
Source: EyeForPharma - August 25, 2020 Category: Pharmaceuticals Authors: Ulrich Neumann Source Type: news

Paying for cell and gene therapy - Is the future already here?
The ascent of cell and gene therapies over the past few years has been astonishing. And their rise looks unstoppable: By 2025, the FDA expects it will be reviewing 10 to 20 of these transformative drugs per year.    But as we listen to affordability concerns from payers, providers and patients, we ’ve also had to ask, perhaps a bit provocatively, how we can afford to pay for this boom in future cures?  " Without solutions to help payers manage the cost, some of our members may make the choice to exclude coverage " , stated insurer CVS Health earlier in the year in its position paper on ...
Source: EyeForPharma - August 25, 2020 Category: Pharmaceuticals Authors: Ulrich Neumann Source Type: news

Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - August 25, 2020 Category: Drugs & Pharmacology Source Type: news

INQOVI (decitabine and cedazuridine) Tablets, FDA Approved for Treatment of Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia, Available at Biologics by McKesson
CARY, N.C., Aug. 24, 2020 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Taiho Oncology, Inc. as a specialty pharmacy provider for INQOVI® (decitabine and cedazuridine) tablets for the treatment of adults with intermedia... (Source: McKesson News)
Source: McKesson News - August 24, 2020 Category: Information Technology Source Type: news

First IOF Skeletal Rare Diseases Academy Awards recognize research excellence
(International Osteoporosis Foundation) Today, at the 2020 World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases, five investigators were awarded the first International Osteoporosis Foundation (IOF) Skeletal Rare Diseases Academy Awards in recognition of the quality of their research related to rare disorders of the skeleton. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 21, 2020 Category: International Medicine & Public Health Source Type: news

Johnson & Johnson Celebrates Role of Scientists In Society with 2020 Dr. Paul Janssen Award for Biomedical Research, Part of the Champions of Science ® Initiative
NEW BRUNSWICK, NJ – August 20, 2020 – Johnson & Johnson today announced Lewis Cantley, Ph.D., of Weill Cornell Medicine and NewYork-Presbyterian as the winner of the 2020 Dr. Paul Janssen Award for Biomedical Research for his incisive research revealing the fundamental aspects of metabolism that have profound implications for the understanding, diagnosis and treatment of human diseases such as cancer and diabetes. “Like all of the past Dr. Paul Janssen Award winners, and indeed like Dr. Janssen himself, Dr. Cantley challenged the status quo, asked provocative questions, and tirelessly followed his cur...
Source: Johnson and Johnson - August 20, 2020 Category: Pharmaceuticals Source Type: news

Why Biliary Atresia Demands Our Respect Why Biliary Atresia Demands Our Respect
Dr William Balistreri explains how interceding quickly can help limit the devastating effects of this rare disease in infants.Medscape Gastroenterology (Source: Medscape Gastroenterology Headlines)
Source: Medscape Gastroenterology Headlines - August 19, 2020 Category: Gastroenterology Tags: Gastroenterology Commentary Source Type: news

Johnson & Johnson to buy biotech Momenta in $6.5bn deal
Tie-up is latest example of big pharma seeking to bolster rare disease portfolio (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - August 19, 2020 Category: Pharmaceuticals Source Type: news

Johnson & Johnson to Acquire Momenta Pharmaceuticals, Inc., Expanding Janssen ’s Leadership in Novel Treatments for Autoimmune Diseases
NEW BRUNSWICK, N.J., August 19, 2020 – Johnson & Johnson (NYSE:JNJ) today announced it has entered into a definitive agreement to acquire 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. This acquisition provides an opportunity for the Janssen Pharmaceutical Companies of Johnson & Johnson to broaden its leadership in immune-mediated diseases and drive further growth through expansion into autoantibody-driven disease. The transaction will include full global rights to nipocalim...
Source: Johnson and Johnson - August 19, 2020 Category: Pharmaceuticals Tags: Our Company Source Type: news

FDA Approves Treatment for Rare Disease Affecting Optic Nerves, Spinal Cord
The FDA has approved a new treatment for neuromyelitis optica spectrum disorder (NMOSD) in adults with a particular antibody. NMOSD is a rare autoimmune disease that mainly affects the optic nerves and spinal cord. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - August 17, 2020 Category: Drugs & Pharmacology Authors: FDA Source Type: news

MD Anderson Leads List of Top-Tier Cancer Hospitals
For the sixth consecutive year, University of Texas MD Anderson Cancer Center in Houston was named the top Adult Cancer Hospital in America by U.S. News & World Report. Several of the top-tier cancer centers on the list have specialty programs for mesothelioma that contributed to their high ranking. The 2020-21 Best Hospitals for Cancer rankings, announced this week, are part of a broader Best Hospitals Honor Roll done annually by U.S. News & World Report. Top five cancer center rankings also included Memorial Sloan Kettering in New York City; Mayo Clinic in Rochester, Minnesota; Johns Hopkins Hospital in Baltimore...
Source: Asbestos and Mesothelioma News - July 29, 2020 Category: Environmental Health Authors: Fran Mannino Source Type: news

Mesothelioma Specialty Centers Rank on 2020 Best Hospitals List
For the sixth consecutive year, University of Texas MD Anderson Cancer Center in Houston was named the top Adult Cancer Hospital in America by U.S. News & World Report. Several of the top-tier cancer centers on the list have specialty programs for mesothelioma that contributed to their high ranking. The 2020-21 Best Hospitals for Cancer rankings, announced this week, are part of a broader Best Hospitals Honor Roll done annually by U.S. News & World Report. Top five cancer center rankings also included Memorial Sloan Kettering in New York City; Mayo Clinic in Rochester, Minnesota; Johns Hopkins Hospital in Baltimore...
Source: Asbestos and Mesothelioma News - July 29, 2020 Category: Environmental Health Authors: Fran Mannino Source Type: news

Meet Dr. David Bartlett, the new leader of AHN Cancer Institute
AHN Cancer Institute is also going to recruit physicians among people that have an existing national reputation in oncology, and focus on unique therapies for rare diseases. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 27, 2020 Category: Biotechnology Authors: Paul J. Gough Source Type: news

9 Meters Biopharma begins patient trials with treatment for rare disease
For CEO John Temperato, it ’s a milestone that at one point seemed a million miles away. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 22, 2020 Category: Pharmaceuticals Authors: Lauren Ohnesorge Source Type: news

More Mesothelioma Cancer Centers Using Tumor Treating Fields
Tumor Treating Fields is one of the newest treatments on the market for pleural mesothelioma cancer patients with metastatic disease. The device is also available to patients with locally advanced cancer who are not candidates for mesothelioma surgery. Clinical trials of the treatment, previously known as NovoTTF-100L, extended the survival of mesothelioma patients by more than six months compared to those receiving only chemotherapy. The device is now called Optune Lua and has been available outside of clinical trials for over a year. More physicians and treatment centers are becoming certified to prescribe the treatmen...
Source: Asbestos and Mesothelioma News - July 21, 2020 Category: Environmental Health Authors: Fran Mannino Source Type: news

Rare disease drove two women on a mission for change
Despite having no prior government experience, two women impacted by a rare genetic disease called FCS helped draft a congressional bill that aims to ensure that rare disease experts and patients are more involved in the FDA drug review process. (Source: CNN.com - Health)
Source: CNN.com - Health - July 19, 2020 Category: Consumer Health News Source Type: news

CZI awards $1.3 million to support the global rare disease community
(Chan Zuckerberg Initiative) The Chan Zuckerberg Initiative is providing $1.3 million in grant funding for rare disease organizations NORD and EURORDIS to support their hundreds of member organizations across the globe. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 16, 2020 Category: International Medicine & Public Health Source Type: news

R & D Focus: 5 things to know about the biopharmaceutical research ecosystem
America ’s biopharmaceutical companies are at the heart of a robust research and development (R&D) ecosystem that develops more innovative medicines than any other country in the world. In recent years, rapid advances in scientific discovery have ushered in a new era of medicine, transforming our ability to treat, and in some cases cure, some of the most challenging diseases, including cancer, rare diseases and autoimmune conditions. These advancements are due to the productivity of the United States ’ biomedical research ecosystem, which is sustained by a policy framework that is designed to support and ad...
Source: The Catalyst - July 13, 2020 Category: Pharmaceuticals Authors: Jocelyn Ulrich Tags: Research and Development Intellectual Property & D Focus Coronavirus Source Type: news

This Startup Might Finally Cure Sickle Cell Disease —After A Century Of Racist Neglect
The painful blood disorder, which mostly affects Black people, is just one of thousands of rare diseases that could be cured by Beam Therapeutics' revolutionary gene editing technology. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 10, 2020 Category: Pharmaceuticals Authors: Katie Jennings, Forbes Staff Tags: Healthcare /healthcare Innovation /innovation Science /science Business /business Leadership /leadership Forbes Daily Cover Stories /daily-cover-stories Editors' Pick editors-pick Source Type: news

Unraveling the Constrictions of Parsonage-Turner Syndrome
Parsonage-Turner Syndrome (PTS), also known as neuralgic amyotrophy, is a rare disease that can be challenging to recognize, leading to delays in diagnosis and impeding physicians’ ability to study the disease and conduct clinical trials. This webinar is intended to raise awareness about the disease, improve recognition and differential diagnosis, and support patients seeking information about their condition. (Source: Orthogate - Latest News)
Source: Orthogate - Latest News - July 8, 2020 Category: Orthopaedics Tags: Featured CME Courses Events Source Type: news

‘It Will Consume Your Life’: 4 Families Take On Rare Diseases
Confronted by illnesses that most scientists overlook, these families had to work out their own approaches to find treatments. (Source: NYT Health)
Source: NYT Health - July 8, 2020 Category: Consumer Health News Authors: Gina Kolata Tags: Rare and Orphan Diseases Genetics and Heredity Research Rett ' s Syndrome NGLY1 Drugs (Pharmaceuticals) Children and Childhood Clinical Trials Parenting Gerstmann-Straussler-Scheinker Syndrome Jansen s Metaphyseal Chondrodysplasi Source Type: news

Trailblazing study discovers novel genetic causes of rare diseases, leading to improved diagnosis and better patient care
A research programme pioneering the use of whole genome sequencing in the NHS has diagnosed hundreds of patients and discovered new genetic causes of disease. (Source: University of Bristol news)
Source: University of Bristol news - July 2, 2020 Category: Universities & Medical Training Tags: Health, Research; Faculty of Biomedical Sciences, School of Cellular and Molecular Medicine, Faculty of Health Sciences, Bristol Medical School, Faculty of Health Sciences, Translational Health Sciences; Press Release Source Type: news

The road to a 'miracle drug': How Ultragenyx and partners found their way to the newest rare disease treatment
The FDA on Tuesday approved the drug, a liquid oil branded as Dojolvi, to treat genetic fatty acid oxidation disorders that can kill babies in their sleep. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 1, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

Blueprint Medicines Announces Submission of New Drug Application to FDA for Pralsetinib for the Treatment of Advanced RET Mutant and RET Fusion-Positive Thyroid Cancers
CAMBRIDGE, Mass., July 1, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced the... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - July 1, 2020 Category: Drugs & Pharmacology Source Type: news

North Bay rare disease drug developer wins 2nd drug approval in 2 weeks
Ultragenyx Pharmaceutical Inc. won its second drug approval in two weeks — and a month ahead of schedule — as regulators greenlighted a treatment against a family of debilitating and potentially deadly genetic disease. The Novato-based company (NASDAQ: RARE) said a 500-milliliter bottle of the drug to treat long-chain fatty acid oxidation disorders will cost $4,875 wholesale, or an average net price of $138,000 per patient per year. Because the disorders can occur in newborns, Ultragenyx said… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 1, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

FDA Approves Dojolvi (triheptanoin) for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
NOVATO, Calif., June 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 30, 2020 Category: Drugs & Pharmacology Source Type: news

2 boys' deaths halt S.F. company's rare disease gene therapy trial
Two boys died in a critical gene therapy trial against a genetic disease, forcing the company behind the therapy and regulators to halt the study. Audentes Therapeutics Inc., a San Francisco company that was sold in January to Astellas Pharma of Japan for $3 billion, said both boys with X-linked myotubular myopathy had been given a higher dose of the therapy and had pre-existing liver problems. The news comes as gene therapies face question s as well as a pivotal moment. BioMarin Pharmaceutical… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 29, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

2 boys' deaths halt S.F. company's rare disease gene therapy trial
Two boys died in a critical gene therapy trial against a genetic disease, forcing the company behind the therapy and regulators to halt the study. Audentes Therapeutics Inc., a San Francisco company that was sold in January to Astellas Pharma of Japan for $3 billion, said both boys with X-linked myotubular myopathy had been given a higher dose of the therapy and had pre-existing liver problems. The news comes as gene therapies face question s as well as a pivotal moment. BioMarin Pharmaceutical… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 29, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

XPOVIO (selinexor), FDA Approved for Treatment of Relapsed or Refractory Diffuse Large B-cell Lymphoma (DLBCL), Available at Biologics by McKesson
CARY, N.C., June 29, 2020 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology, rare diseases and other complex therapeutic areas, was selected by Karyopharm Therapeutics, Inc. as a specialty pharmacy provider for XPOVIO® (selinexor) for the treatment of adult patients w... (Source: McKesson News)
Source: McKesson News - June 29, 2020 Category: Information Technology Source Type: news

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the Company has completed the submission of a rolling New Drug Application... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - June 26, 2020 Category: Drugs & Pharmacology Source Type: news

FDA Approves Fintepla (fenfluramine) for the Treatment of Seizures Associated with Dravet Syndrome
EMERYVILLE, Calif., June 25, 2020 (GLOBE NEWSWIRE) -- Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved Fintepla... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 26, 2020 Category: Drugs & Pharmacology Source Type: news

Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO)
NOVATO, Calif. and TOKYO, June 18, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 19, 2020 Category: Drugs & Pharmacology Source Type: news

Subcutaneous Daratumumab Combination Resulted in Deep and Rapid Hematologic Responses and Improved Clinical Outcomes in the Treatment of Patients with Newly Diagnosed Light Chain (AL) Amyloidosis
RARITAN, NJ, June 13, 2020 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today results from the first randomized Phase 3 study investigating subcutaneous daratumumab[i] in the treatment of patients with newly diagnosed light chain (AL) amyloidosis, a rare and potentially fatal disease.[1],[2] The data demonstrated subcutaneous daratumumab in combination with cyclophosphamide, bortezomib, and dexamethasone (D-CyBorD) resulted in a higher hematologic complete response rate (CR), (53 percent vs. 18 percent [P
Source: Johnson and Johnson - June 15, 2020 Category: Pharmaceuticals Tags: Innovation Source Type: news