NIDCR's Spring 2021 E-Newsletter
Having trouble viewing this email? View it as a Web page. NIDCR's Spring 2021 E-Newsletter In this issue: NIDCR News Funding Opportunities & Related Notices NIH/HHS News Subscribe to NICDR News Science Advances   Grantee News   NIDCR News NIDCR & NIH Stand Against Structural Racism NIDCR Director Rena D’Souza, DDS, MS, PhD, said in a statement that there is no place for structural racism in biomedical research, echoing remarks from NIH Director Francis Collins, MD, PhD, in his announcement of a new NIH ...
Source: NIDCR Science News - April 7, 2021 Category: Dentistry Source Type: news

A new mouse model gave surprising findings about Folling Disease
(The University of Bergen) A new study from the University in Bergen may shed light on the comorbidities found in adults suffering from this rare disease. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - April 7, 2021 Category: Biology Source Type: news

India notifies first policy on rare diseases
In the new policy, formulated after detailed stakeholder consultations, 'rare diseases' have not been defined but classified into three groups based on expert opinion. (Source: The Economic Times)
Source: The Economic Times - April 1, 2021 Category: Consumer Health News Source Type: news

Rapid Genome Sequencing Can Save Babies With Rare Diseases, If They Can Get It
Undiagnosed genetic diseases take a serious physical and emotional toll on families. Rapid genome sequencing can provide answers and guide treatment decisions, but so far, insurance companies have been reluctant to pay. That's beginning to change. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - March 31, 2021 Category: Pharmaceuticals Authors: Caroline Seydel, Contributor Tags: Healthcare /healthcare Innovation /innovation Source Type: news

Are there common walking gait characteristics in patients diagnosed with late-onset Pompe disease? - Starbuck C, Reay J, Silk E, Roberts M, Hendriksz C, Jones R.
Late-onset Pompe disease (LOPD) is a rare disease, defined as a progressive accumulation of lysosomal glycogen resulting in muscle weakness and respiratory problems. Anecdotally, individuals often have difficulties walking, yet, there is no three-dimension... (Source: SafetyLit)
Source: SafetyLit - March 20, 2021 Category: International Medicine & Public Health Tags: Ergonomics, Human Factors, Anthropometrics, Physiology Source Type: news

'Germ Hunters' discover rare disease in rural Alberta
(University of Alberta Faculty of Medicine& Dentistry) A rare lung disease that is linked to bats has made Alberta home, according to new research led by scientists at Alberta Precision Laboratories and the University of Alberta. The team of infectious disease experts confirmed that histoplasmosis--a fungal infection transmitted through bat and bird droppings--is now found in Alberta. Their study extends the known range of the disease much farther northwest from its traditional home in the central United States and parts of southern Ontario and Quebec. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - March 18, 2021 Category: Infectious Diseases Source Type: news

Individuals with Barth Syndrome Chart New Path for the Review of Drugs
Barth Syndrome Foundation’s recent listening session with FDA sheds light on one rare disease community’s acceptance of less certainty of treatment benefits(PRWeb March 17, 2021)Read the full story at https://www.prweb.com/releases/individuals_with_barth_syndrome_chart_new_path_for_the_review_of_drugs/prweb17802694.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 17, 2021 Category: Pharmaceuticals Source Type: news

PCORI approves $48.5 million to fund new research and projects to speed uptake of evidence into practice
(Patient-Centered Outcomes Research Institute) PCORI Board approves $48.5 million to fund new comparative clinical effectiveness research on second-line medications for Type 2 diabetes and several rare diseases. The Board approved another $5.8 million for four projects to facilitate adoption of useful findings from completed research in clinical care. PCORI also will issue new funding announcements on maternal health, urinary incontinence and mental health among people with intellectual and developmental disabilities. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 16, 2021 Category: International Medicine & Public Health Source Type: news

Stanford gene-editing startup raises $150M for sickle cell clinical trial, expansion into rare diseases
The South San Francisco company's early work at Stanford — as well as the work of a critical partner — was backed by grants from California's stem cell research funding agency CIRM. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 15, 2021 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

What price a child's life? India's quest to make rare disease drugs affordable
Parents whose only hope was finding foreign sponsorship or a clinical trial are now looking for homegrown breakthroughsFor three years, Vidya tried to find the cause of her son ’s recurrent fevers and low cognitive development. When she found out, she was devastated.Vineeth, 10, has an incurable illness – mucopolysaccharidosis type 2 – that affects his organs. Afflicting just one in a million, the enzyme-replacement medication that can help stop the illness getting any worse costs £100,000 a year, far beyond the reach of even a wealthier Indian parent.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - March 15, 2021 Category: Science Authors: Priti Salian Tags: Global development Global health India Children's health Medical research Pharmaceuticals industry Genetics Biology Business Science Society South and Central Asia World news Source Type: news

FOTIVDA (tivozanib), FDA Approved for Treatment of Relapsed/Refractory Advanced Renal Cell Carcinoma, Available at Biologics by McKesson
CARY, N.C., March 12, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, was selected by AVEO Oncology as a specialty pharmacy provider for FOTIVDA® (tivozanib) for the treatment of adult patients with relapsed or refractory advanced renal ... (Source: McKesson News)
Source: McKesson News - March 12, 2021 Category: Information Technology Source Type: news

Teenager is the only person in the world with unique combination of rare illnesses
A BRITISH teenager with a one-off combination of extremely rare diseases is now working to raise awareness of her conditions. (Source: Daily Express - Health)
Source: Daily Express - Health - March 8, 2021 Category: Consumer Health News Source Type: news

Strongbridge Biopharma files new drug application with FDA for rare disease therapy
The new drug candidate targets a condition that afflicts about 8,000 people in the United States. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 3, 2021 Category: Pharmaceuticals Authors: John George Source Type: news

Strongbridge Biopharma plc Announces Submission of New Drug Application for Recorlev (levoketoconazole) for the Treatment of Endogenous Cushing ’s Syndrome to the FDA
DUBLIN, Ireland and TREVOSE, Pa., March 02, 2021 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - March 2, 2021 Category: Drugs & Pharmacology Source Type: news

Rare Disease Day 2021: FDA Shows Sustained Support of Rare Disease Product Development During the Public Health Emergency
As the FDA focuses on the COVID-19 pandemic, the agency also remains dedicated to its crucial role in development of treatments for rare diseases. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - March 1, 2021 Category: Drugs & Pharmacology Authors: FDA Source Type: news

India can lead the charge for discovery of drugs for other rare diseases: NITI Aayog's Dr. Vinod Paul
Speaking specifically about orphan drugs, Paul said that with the support of the government, remedies for rare diseases can be discovered in India, which can then benefit the entire world. (Source: The Economic Times)
Source: The Economic Times - March 1, 2021 Category: Consumer Health News Source Type: news

Tackling deadly newborn disease, BridgeBio affiliate wins FDA drug approval
The BridgeBio model is designed to move drugs tackling rare diseases with a well-defined genetic basis quickly and efficiently to approval. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - February 26, 2021 Category: American Health Authors: Ron Leuty Source Type: news

Tackling deadly newborn disease, BridgeBio affiliate wins FDA drug approval
The BridgeBio model is designed to move drugs tackling rare diseases with a well-defined genetic basis quickly and efficiently to approval. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 26, 2021 Category: Biotechnology Authors: Ron Leuty Source Type: news

Translating passion into execution: Cure Rare Disease's founder on the business of biotech
When a career path chooses you in the form of a pressing personal cause, passion and commitment must soon be followed by execution. And that means schooling up — quickly. I had heard the call of entrepreneurship early. I had a new Harvard Business School degree in hand at the same time my brother’s health was declining steeply due to Duchenne muscular dystrophy. His fatal degenerative neuromuscular condition wouldn't wait, and the lack of viable option s in the pipeline for his gene mutation required entrepreneurial thinking. The science would come. I was in Boston, after all, immersed in bioscience. And it has...
Source: bizjournals.com Health Care:Biotechnology headlines - February 26, 2021 Category: Biotechnology Authors: Rich Horgan Source Type: news

FDA Approves Amondys 45 (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Amondys 45... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - February 25, 2021 Category: Drugs & Pharmacology Source Type: news

World-leading children's hospitals partner to find new treatments for pediatric diseases
(University College London) Ahead of Rare Disease Day (28 February), four paediatric hospitals -- Boston Children's Hospital; UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital (London); the Murdoch Children's Research Institute with The Royal Children's Hospital (Melbourne); and The Hospital for Sick Children (SickKids) in Toronto -- are launching IPCHiP, a new collaborative to decipher paediatric illnesses, including rare diseases, and find better treatments. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - February 25, 2021 Category: International Medicine & Public Health Source Type: news

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Positive Topline Results from BRIGHT Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa 2 mg/kg every Four Weeks for Treatment of Fabry Disease
Study achieved key objectives for safety, efficacy and pharmacokinetics After completion of the study, all patients enrolled in an extension study pegunigalsidase alfa (PRX-102) provided coverage to patients for the entire 4-week period in treated pa... Biopharmaceuticals Protalix BioTherapeutics, Chiesi , Fabry disease, pegunigalsidase alfa (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 23, 2021 Category: Pharmaceuticals Source Type: news

FDA Fast-Tracks Mesothelioma Vaccine Development
The U.S. Food and Drug Administration has granted Fast-Track Designation this week to ONCOS-102, an immunotherapy vaccine that targets malignant mesothelioma and other hard-to-treat tumors. This designation is expected to expedite the regulatory approval process. The vaccine’s developer, Targovax, a small biotech company that focuses on oncolytic viruses, sees this as an early endorsement from the FDA. “Yes, this opens the door for us,” Dr. Magnus Jaderberg, chief medical officer of Targovax, told The Mesothelioma Center at Asbestos.com. “It validates what the FDA believes is a potentially promising...
Source: Asbestos and Mesothelioma News - February 19, 2021 Category: Environmental Health Authors: Amy Edel Source Type: news

Bringing innovation to rare disease patients in Europe
Why is Biogen focusing on rare diseases?  About 6,000 different rare diseases have been identified to date and these are estimated to affect 30 million people across Europe. It is essential that we collaborate across the medical community, industry, and governments to develop new therapies, explore new technologies and ensure equitable access to treatment. We must innovate across every aspect of treatment to transform the lives of people in these underserved communities.  At Biogen, we have shown that pioneering in neuroscience can change the course of treatment for certain rare diseases. We have transformed the ...
Source: EyeForPharma - February 17, 2021 Category: Pharmaceuticals Authors: Izzy Gladstone Source Type: news

Large-scale study finds genetic testing technology falsely detects very rare variants
(University of Exeter) A technology that is widely used by commercial genetic testing companies is 'extremely unreliable' in detecting very rare variants, meaning results suggesting individuals carry rare disease-causing genetic variants are usually wrong, according to new research published in the BMJ. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 15, 2021 Category: International Medicine & Public Health Source Type: news

#Icare4Rare campaign launches to raise awareness ahead of Rare Disease Week 2021
For many of the 30 million people affected by rare disease in the EU, lack of effective treatments and discrepancies in access and diagnosis are still common issues. Strong political will, innovation and further cooperation between countries remain vital to improve outcomes and transform their quality of life.To help raise awareness of rare disease, Reuters Events, Biogen and EU40 have collaborated to launch the #Icare4rare campaign to give prominence to Rare Disease Week 2021.Andrea Corazza, Head of Brussels Liaison Office, Public Policy& Government Affairs, Biogen, says: “With this campaign, we want to help put...
Source: EyeForPharma - February 11, 2021 Category: Pharmaceuticals Authors: Izzy Gladstone Source Type: news

UCLA Health receives $29 million gift to establish center in precision genomic medicine
UCLA has received a $29 million gift to establish a center where scientists and physicians will work side by side to examine the role of genetics in disease and develop therapies that improve patients ’ lives.The gift creates the Dr. Allen and Charlotte Ginsburg Center for Precision Genomic Medicine. The new center will build on UCLA ’s efforts in precision health to leverage large data sets and innovative genomic technologies such as CRISPR engineering to improve diagnosis and treatment of a wide variety of genetic disorders. They include both rare diseases and more common illnesses such as cardiovascular dise...
Source: UCLA Newsroom: Health Sciences - February 11, 2021 Category: Universities & Medical Training Source Type: news

Rare Disease Therapy Development and Access Remain Top FDA Priorities During COVID-19
FDA ’s work to advance treatments for rare diseases and helping ensure continuity of care for people with rare diseases remain top priorities during COVID-19. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - February 4, 2021 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Finalise policy on rare diseases by March 31: High Court to Centre
Justice Singh said she was issuing the direction to finalise the policy and operationalise the digital platform by March 31 as an affidavit filed by the Health Ministry said that both things were only "likely" to be done by March 31. (Source: The Economic Times)
Source: The Economic Times - January 28, 2021 Category: Consumer Health News Source Type: news

Partners in crime: genetic collaborator may influence severity of the rare disease, NGLY1
(University of Utah Health) In 2012, four-year-old Bertrand Might became the first-ever patient diagnosed with a rare genetic disorder called N-glycanase (NGLY1) deficiency. The discovery of this condition and Bertrand's diagnosis allowed doctors to look for other children with the same genetic defect. Since then, more than 60 additional patients have been found. Clement Chow, a University of Utah geneticist is determined to find what's going on. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 26, 2021 Category: International Medicine & Public Health Source Type: news

ORGOVYX (relugolix), the First FDA-Approved Oral Gonadotropin-Releasing Hormone (GnRH) Receptor Antagonist for Advanced Prostate Cancer, Available at Biologics by McKesson
CARY, N.C., Jan. 21, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Myovant Sciences as a specialty pharmacy provider for ORGOVYXTM (relugolix) for the treatment of advanced prostate cancer. (Source: McKesson News)
Source: McKesson News - January 21, 2021 Category: Information Technology Source Type: news

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