REZUROCK (belumosudil) tablets, FDA Approved for Treatment of Chronic Graft-Versus-Host Disease, Available at Biologics by McKesson
CARY, N.C., July 30, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, today announced that it has been selected by Kadmon (Nasdaq: KDMN) as a specialty pharmacy provider for REZUROCK™ (belumosudil) for the treatment of adult and pediatric... (Source: McKesson News)
Source: McKesson News - July 30, 2021 Category: Information Technology Source Type: news

FDA Grants Orphan Drug Designation to Novel Mesothelioma Treatment
The U.S. Food and Drug Administration has granted orphan drug designation to ONCOFID-P for the treatment of malignant pleural mesothelioma, a step toward another potential therapy option for patients. ONCOFID-P is an innovative drug conjugate already in advanced clinical development for the treatment of invasive bladder cancer. The mesothelioma designation is based on preclinical data showing high antitumor activity in laboratory and mice models. Fidia Farmaceutici, a multinational research company based in Italy, announced the latest FDA designation, which is designed to encourage the development of novel drugs, par...
Source: Asbestos and Mesothelioma News - July 28, 2021 Category: Environmental Health Authors: Fran Mannino Source Type: news

Researcher Hopes to Improve Tumor Treating Fields for Mesothelioma
Dr. Maurizio D’Incalci already has seen Tumor Treating Fields working for patients with malignant pleural mesothelioma, and applauded the U.S. Food and Drug Administration’s approval of the treatment in 2019. He also knows the therapy is only scratching the surface of its vast potential and could be even more effective. He wants to help improve it. D’Incalci, a heralded biomedical science professor at Humanitas University in Milan, Italy, has begun a study exploring various drug combinations that could have a positive, synergistic effect with Tumor Treating Fields for mesothelioma. “There m...
Source: Asbestos and Mesothelioma News - July 27, 2021 Category: Environmental Health Authors: Fran Mannino Source Type: news

What companies can learn about working together to find solutions for the rare disease community
One of the most significant challenges facing the rare disease community is the length of time it can take for a patient to receive an accurate diagnosis of their disease. The Journal of Rare Disorders reports that on average it takes a rare disease patient 4.8 years, seeing 7.3 specialists to receive an accurate diagnosis, and some patients never receive one. Recognizing this challenge, The Manton Center for Orphan Disease Research at Boston Children's Hospital (BCH) and Alexion Pharmaceuticals… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 21, 2021 Category: Biotechnology Authors: Alexion Pharmaceuticals Source Type: news

NIH-funded study finds gene therapy may restore missing enzyme in rare disease
Results provide hope for children with aromatic L-amino acid decarboxylase deficiency. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - July 16, 2021 Category: American Health Source Type: news

NIH-funded study finds gene therapy may restore missing enzyme in rare disease
(NIH/National Institute of Neurological Disorders and Stroke) A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 16, 2021 Category: International Medicine & Public Health Source Type: news

Researcher creates cell lines to help treat mitochondrial diseases in children
(Virginia Tech) The mitochondrion has garnered quite the reputation for its role as the " powerhouse of the cell. " These tiny, but mighty organelles play various life-sustaining roles, from powering our own cells and organs to fueling chemical and biological processes. But when they aren't working properly, a number of rare diseases can occur. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 15, 2021 Category: International Medicine & Public Health Source Type: news

Dying patients with rare diseases struggle to get experimental therapies
Getting expanded access to unproven therapies for rare diseases is more difficult than for more common illnesses, such as cancer, experts say. (Source: Reuters: Health)
Source: Reuters: Health - July 10, 2021 Category: Consumer Health News Source Type: news

Namibia: Living With Pott's Disease
[Namibian] WHILE the world is battling the deadly Covid-19 pandemic, 36-year-old Joseph Haikali is fighting a rare disease that causes his joints and knees to continuously keep swelling. (Source: AllAfrica News: Tuberculosis)
Source: AllAfrica News: Tuberculosis - July 7, 2021 Category: Infectious Diseases Source Type: news

Scientific Journeys: From basic researcher to leader in autoimmunity
Lisa Rider, M.D., discussed her path from medical school to a career in translational research for myositis, a rare disease. (read more) (Source: Environmental Factor - NIEHS Newsletter)
Source: Environmental Factor - NIEHS Newsletter - July 2, 2021 Category: Environmental Health Source Type: news

NIDCR's Summer 2021 E-Newsletter
Having trouble viewing this email? View it as a Web page. NIDCR's Summer 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 to Release Report on Oral Health in America As a 20-year follow-up to the seminal Oral Health in America: A Report of the Surgeon General, NIDCR will release Oral Health in America: Advances and Challenges in the fall of 2021. The report will illuminate new directions...
Source: NIDCR Science News - July 1, 2021 Category: Dentistry Source Type: news

Meeting needs in rare disease takes more than medicine
The Covid-19 pandemic has had a major effect on all of our lives. For many, the pandemic and ensuing social distancing measures have increased feelings of loneliness, stress, worry and other strong emotions putting mental health in the spotlight. As a result, many business and community leaders are placing a greater emphasis on emotional health and well-being. While awareness of mental health has been brought to the forefront because of the pandemic, stigmas, financial expense and limited access… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - June 30, 2021 Category: American Health Authors: Alex Schuman Source Type: news

Meeting needs in rare disease takes more than medicine
The Covid-19 pandemic has had a major effect on all of our lives. For many, the pandemic and ensuing social distancing measures have increased feelings of loneliness, stress, worry and other strong emotions putting mental health in the spotlight. As a result, many business and community leaders are placing a greater emphasis on emotional health and well-being. While awareness of mental health has been brought to the forefront because of the pandemic, stigmas, financial expense and limited access… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 29, 2021 Category: Biotechnology Authors: Alex Schuman Source Type: news

GPS Vaccine Plus Opdivo Shows Promise for Pleural Mesothelioma
A novel, targeted cancer vaccine combined with the immunotherapy drug Opdivo has shown surprising efficacy for mesothelioma patients whose disease no longer responds to standard chemotherapy. In a phase I clinical trial at Memorial Sloan Kettering Cancer Center, the galinpepimut-S vaccine, also known as GPS, has produced an impressive, anti-tumor synergy with Opdivo, based upon early data. GPS targets the WT1 protein, which is expressed in high levels of various cancers but rarely seen in normal adult cells. The protein has been especially resistant to standard chemotherapy. The vaccine works by inducing a powerfu...
Source: Asbestos and Mesothelioma News - June 29, 2021 Category: Environmental Health Authors: Fran Mannino Source Type: news

3 things that support robust biopharmaceutical research and development
Rapid advances in scientific discovery have ushered in a new era of medicine, transforming our ability to treat, and in some cases cure, many of the most challenging diseases, including cancer, rare diseases and autoimmune conditions.  (Source: The Catalyst)
Source: The Catalyst - June 29, 2021 Category: Pharmaceuticals Authors: Richard Moscicki, M.D. Tags: Research and Development PDUFA & D Focus Source Type: news

Rockville's Vigene Biosciences closes $292M deal to be acquired
Charles River Laboratories International Inc. (NYSE: CRL) said Tuesday morning it had completed its acquisition of Vigene Biosciences Inc., a Rockville firm that helps gene therapy and vaccine companies develop and manufacture products for rare diseases. Massachusetts-based Charles River paid $292.5 million for Vigene, and it said the purchase price could include up to $57.5 million in additional payments contingent on future performance. When the deal was first announced in May, Charles River… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 29, 2021 Category: Pharmaceuticals Authors: Drew Hansen Source Type: news

Rockville's Vigene Biosciences closes $292M deal to be acquired
The firm helps gene therapy and vaccine companies develop and manufacture products for rare diseases. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 29, 2021 Category: Pharmaceuticals Authors: Drew Hansen Source Type: news

CRISPR Therapy Fights Rare Disease Where Protein Clogs Organs
TUESDAY, June 29, 2021 -- Early research suggests that CRISPR gene-editing technology may some day lead to dramatic relief for patients struggling with amyloidosis, a rare but serious disease that can trigger organ failure. " There are many... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - June 29, 2021 Category: General Medicine Source Type: news

CRISPR Therapy Fights Rare Disease Where Protein Clogs Organs
Title: CRISPR Therapy Fights Rare Disease Where Protein Clogs OrgansCategory: Health NewsCreated: 6/29/2021 12:00:00 AMLast Editorial Review: 6/29/2021 12:00:00 AM (Source: MedicineNet Arthritis General)
Source: MedicineNet Arthritis General - June 29, 2021 Category: Rheumatology Source Type: news

What Types of New Therapies are Available for Cystic Fibrosis?
Discussion Cystic fibrosis (CF) was identified in 1938 by Dr. Dorothy Andersen who described 49 patients with pancreatic insufficiency. Since that time significant achievements in the knowledge about the disease and treatments for patients have changed the mortality from a few months to patients living into middle adulthood or even later. Quality of life for patients and their families and friends has also markedly improved. CF is the classic Mendelian autosomal recessive genetic disorder which is a worldwide disorder but affects people of north European ancestry more often where the main mutation is more common. There are...
Source: PediatricEducation.org - June 28, 2021 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

'Genomics of Rare Diseases. Understanding Disease Genetics Using Genomic Approaches'
(Baylor College of Medicine) " Genomics of Rare Diseases " offers readers a broad understanding of current knowledge on rare diseases from a genomics perspective. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 25, 2021 Category: International Medicine & Public Health Source Type: news

Inclusion programs need a foundation of listening: 5 steps to promote active, empathetic listener interactions
A study published in the Harvard Business Review, identified the characteristics that make someone an outstanding listener. It found that good listeners make the other person feel supported through a two-way dialog, rather than a one-way speaker versus listener interaction. As a global biopharmaceutical company focused on developing life-changing medicines for people living with rare diseases and devastating conditions, Alexion recognizes that actively listening to all our stakeholders can lead… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - June 21, 2021 Category: American Health Authors: Uzair Qadeer Source Type: news

TRUSELTIQ (infigratinib), FDA Approved for Treatment of Cholangiocarcinoma, Available at Biologics by McKesson
CARY, N.C., June 21, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by QED Therapeutics, an affiliate of BridgeBio Pharma, Inc. (BridgeBio) and Helsinn Group, as a specialty pharmacy provider for TRUSELTIQTM (infigrati... (Source: McKesson News)
Source: McKesson News - June 21, 2021 Category: Information Technology Source Type: news

Orphazyme Provides Regulatory Update from FDA on Arimoclomol for Niemann-Pick Disease Type C
Copenhagen – June 18, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today announced it has received a... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - June 18, 2021 Category: Drugs & Pharmacology Source Type: news

LUMAKRAS (sotorasib), FDA Approved for Treatment of KRAS G12C-mutated Locally Advanced or Metastatic Non-small Cell Lung Cancer, Available at Biologics by McKesson
CARY, N.C., June 11, 2021 — Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Amgen Oncology as a specialty pharmacy provider for LUMAKRASTM (sotorasib) for the treatment of patients with KRAS G12C-mutated locally advanced ... (Source: McKesson News)
Source: McKesson News - June 11, 2021 Category: Information Technology Source Type: news

Alexion exec to become AstraZeneca's CFO on closing of $39 billion acquisition
AstraZeneca PLC has appointed Dr. Aradhana Sarin as an executive director and chief financial officer conditional upon closing of AstraZeneca ’s acquisition of Boston-based Alexion Pharmaceuticals, Inc., where she is currently executive vice president and CFO. Astra Zeneca in December agreed to purchase Alexion, a global rare diseases biopharmaceutical company, for $39 billion in cash and stock. The sale is expected to close in the th ird quarter of 2021. Sarin will relocate from the United States… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 7, 2021 Category: Pharmaceuticals Authors: Anne Stych Source Type: news

Up close and personal – The Dr. Kevin Williams story
From a young age, Dr. Kevin Williams looked to his father as a role model. Back in 1952, his father became the third African American physician in Baton Rouge, Louisiana, despite barriers and discrimination faced by Black professionals in the medical field.   “It was a different time,” says Kevin. “When my father first opened his practice, none of the hospitals would give him admitting privileges.”  At his office, Kevin ’s father saw patients on a first come, first served basis. Patients in the area were so eager to see him that they started lining up at 4 o’clock eac...
Source: EyeForPharma - June 7, 2021 Category: Pharmaceuticals Authors: Jill Donahue Source Type: news

Up close and personal – The Dr. Kevin Williams story
From a young age, Dr. Kevin Williams looked to his father as a role model. Back in 1952, his father became the third African American physician in Baton Rouge, Louisiana, despite barriers and discrimination faced by Black professionals in the medical field.   “It was a different time,” says Kevin. “When my father first opened his practice, none of the hospitals would give him admitting privileges.”  At his office, Kevin ’s father saw patients on a first come, first served basis. Patients in the area were so eager to see him that they started lining up at 4 o’clock eac...
Source: EyeForPharma - June 7, 2021 Category: Pharmaceuticals Authors: Jill Donahue Source Type: news

How putting the patient at the center of every decision drives one company ’s culture
Most people have gone through the experience of having a health concern and not feeling like it ’s being handled as well as it could be. For patients with rare diseases, this experience is even more common, as they navigate complex health care journeys involving multiple physicians and other health care professionals, insurance providers, caregivers, family members, employers and patient org anizations. According to the National Institutes of Health, there are an estimated 30 million people in… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 3, 2021 Category: Biotechnology Authors: Rana Strellis, SVP, Global Culture and Corporate Social Responsibility, and Sonia Singhvi, Head of Culture Source Type: news

Treatabolome project designed to shorten diagnosis-to-treatment time for patients with rare diseases
(IOS Press) The Treatabolome project is a research initiative to develop a freely available, interoperable online platform to disseminate rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise to reduce treatment delays. This initiative is highly relevant to neuromuscular disorders as they are rare diseases by definition. In this special issue of the Journal of Neuromuscular Diseases, experts contribute Treatabolome-feeding systematic literature reviews on rare neurological and neuromuscular disorders. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 1, 2021 Category: International Medicine & Public Health Source Type: news

Unprecedented data sharing driving new rare disease diagnoses in Europe
(Center for Genomic Regulation) Massive data sharing across Europe is boosting efforts to diagnose rare diseases for individuals whose case remains unsolved even after going through advanced expert clinical settings. A new method developed by an international consrortium has resulted in 255 new diagnoses, which is the 'tip of the icerberg'. The findings are an important first step for a European-wide system to facilitate the diagnosis rare diseases, which can be a long and arduous process. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - June 1, 2021 Category: International Medicine & Public Health Source Type: news

Trust is key for the parents of children with rare diseases to live without anxiety
(Universitat Oberta de Catalunya (UOC)) The parents of children with rare diseases face exceptional circumstances which influence their role as parents. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - May 27, 2021 Category: International Medicine & Public Health Source Type: news

Updated Data Demonstrate Significant Improvement in Hematologic Complete Response with DARZALEX FASPRO ® (daratumumab and hyaluronidase-fihj) in Patients with Newly Diagnosed Light Chain (AL) Amyloidosis
RARITAN, N.J., May 26, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced updated results from the Phase 3 ANDROMEDA study, which evaluated DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) for the treatment of patients with newly diagnosed light chain (AL) amyloidosis, a rare blood cell disorder associated with the deterioration of vital organs, most notably the heart, kidneys and liver.[1] Longer-term results from a median follow-up of 20.3 months showed rates of hematologic complete response (hemCR) remained significantly higher in patients treated with DARZALEX FASPRO®...
Source: Johnson and Johnson - May 26, 2021 Category: Pharmaceuticals Tags: Innovation Source Type: news

Posttraumatic bronchobiliary fistulae due to foreign body remnants after  a road traffic injury: a case report - Seyed-Alagheband SA, Shahmoradi MK, Shekouhi R.
BACKGROUND: Bronchobiliary fistula is an extremely rare disease that involves abnormal communication between a hepatic segment and bronchial tree. It is mostly caused by untreated hydatid cyst, liver abscess, iatrogenic stenosis, and, rarely, trauma. CASE ... (Source: SafetyLit)
Source: SafetyLit - May 24, 2021 Category: International Medicine & Public Health Tags: Economics of Injury and Safety, PTSD, Injury Outcomes Source Type: news

Including the caregiver in access submissions
While pharma now understands the merit of placing the patient at the centre of all it does, the industry typically gives too little consideration to another important stakeholder - the caregiver. Better products and outcomes are two benefits of doing so. Another is the potential to make a stronger case for a therapy with payers. Because caregivers often prescribe or administer a medicine, they can provide valuable insights to strengthen evidence in access submissions.   “Often, in rare diseases the patient’s abilities are compromised,” says Raquel Cabo, VP of market access and patient services ...
Source: EyeForPharma - May 23, 2021 Category: Pharmaceuticals Authors: Katie Osborne Source Type: news

Strongbridge Biopharma plc Announces U.S. Food & Drug Administration (FDA) Filing Acceptance of New Drug Application (NDA) for Recorlev (levoketoconazole) for the Treatment of Endogenous Cushing ’s Syndrome
DUBLIN, Ireland and TREVOSE, Pa., May 13, 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 - May 17, 2021 Category: Drugs & Pharmacology Source Type: news

Overcoming Challenges in Cell-Based Research
Lessons learned from rare diseases (Source: The Scientist)
Source: The Scientist - May 11, 2021 Category: Science Tags: The Marketplace Source Type: news

LogicBio Therapeutics Strengthens Management Team with Key Appointments
New leadership includes Andrea Paul as general counsel and corporate secretary, and Janice Olson as senior vice president, strategy and portfolio management, bringing extensive experience in gene therapy and rare disease LEXINGTON, Mass., May 10, 2021 -... Regenerative Medicine, Personnel LogicBio Therapeutics, GeneRide (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 10, 2021 Category: Pharmaceuticals Source Type: news

Peninsula drugmaker's rollercoaster week ends with 62% stock drop
An FDA advisory committee by the thinnest of margins recommended that the agency approve the company's rare disease drug. Investors and regulators had questions. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 7, 2021 Category: American Health Authors: Ron Leuty Source Type: news

Peninsula drugmaker's rollercoaster week ends with 62% stock drop
An FDA advisory committee by the thinnest of margins recommended that the agency approve the company's rare disease drug. Investors and regulators had questions. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 7, 2021 Category: Biotechnology Authors: Ron Leuty Source Type: news

Pittsburgh native to represent U.S. in world entrepreneur of the year competition
Gordon J. Vanscoy, chairman and CEO of Pittsburgh-based RareMed Solutions and founder and executive advisor of PANTHERx Rare, will represent the U.S. in the upcoming Ernst& Young LLP  World Entrepreneur of the Year competition in June 2021, according to a news release.  Both companies provide services to people with rare disorders, and PANTHERx Rare is the only “pure rare disease and orphan drug-focused” pharmacy in the country. Vanscoy has more than 30 years of experienc e in developing medical… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 6, 2021 Category: Biotechnology Authors: Julia Mericle Source Type: news

Pittsburgh biotech names Alnylam exec as new chief commercial officer
Pittsburgh-based Krystal Biotech Inc. named a veteran biosciences executive as its new chief commercial officer. Andy Orth will be in charge of launching Krystal’s medicines into the marketplace. He has 25 years of experience in the sector and had been SVP and head of U.S. business at Alnylam Pharmaceuticals, a Cambridge-based biopharmaceutical developer. Krystal (Nasdaq: KRYS) is a Pittsburgh-based compa ny that has a platform that can create gene therapies to treat rare diseases where a gene… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 4, 2021 Category: Biotechnology Authors: Paul J. Gough Source Type: news

Pittsburgh biotech names Alnylam exec as new chief commercial officer
Pittsburgh-based Krystal Biotech Inc. named a veteran biosciences executive as its new chief commercial officer. Andy Orth will be in charge of launching Krystal’s medicines into the marketplace. He has 25 years of experience in the sector and had been SVP and head of U.S. business at Alnylam Pharmaceuticals, a Cambridge-based biopharmaceutical developer. Krystal (Nasdaq: KRYS) is a Pittsburgh-based compa ny that has a platform that can create gene therapies to treat rare diseases where a gene… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 4, 2021 Category: American Health Authors: Paul J. Gough Source Type: news

Towards a treatment for myotonic dystrophy: First 3D model with patient cells
(Institute for Bioengineering of Catalonia (IBEC)) Researchers from the Institute for Bioengineering of Catalonia (IBEC) in collaboration with the INCLIVA Health Research Institute in Valencia, develop the first three-dimensional model for myotonic dystrophy, a rare disease that currently has no cure. The new model combines patient cells and bioengineering techniques and represents a major advance over the use of animals and cell cultures. This new model will help in the design of personalized and more effective treatments, and for drug testing in a much more efficient way. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - May 3, 2021 Category: Biology Source Type: news

Chiesi Global Rare Diseases Announces FDA Approval of Ferriprox (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease
May 1, 2021 Approval is based on demonstrated reduction in liver iron concentration Expanded indications for patients with sickle cell disease or other anemias, as well as thalassemia - BOSTON, May 1, 2021 /PRNewswire/ -- Chiesi Global Rare... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 1, 2021 Category: Drugs & Pharmacology Source Type: news

Science Saturday: Researchers explore personalized approach to anti-obesity medications
Individualized medicine initiatives mainly focus on rare diseases or cancer. Little has been attempted to individualize treatment for noncommunicable chronic diseases such as obesity -- a chronic, relapsing disease, and a primary cause of Type 2 diabetes, fatty liver disease, cardiovascular disease and cancer. There are many obesity interventions, such as diets, devices, surgery and [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - May 1, 2021 Category: Databases & Libraries Source Type: news

Single-cell CRISPR technology deciphers role of chromatin accessibility in cancer
(New York Genome Center) Researchers in the lab of Neville Sanjana, PhD, at the New York Genome Center (NYGC) and New York University (NYU) have developed CRISPR-sciATAC, a novel integrative genetic screening platform that jointly captures CRISPR gene perturbations and single-cell chromatin accessibility genome-wide. The new method harnesses the programmability of the gene editing system CRISPR to knock-out nearly all chromatin-related genes in parallel, offering researchers deeper insights into the role of DNA accessibility in cancer and in rare diseases involving chromatin. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - April 29, 2021 Category: Cancer & Oncology Source Type: news

Protalix BioTherapeutics and Chiesi Global Rare Diseases Receive Complete Response Letter for Pegunigalsidase Alfa from FDA
CARMIEL, Israel and BOSTON, April 28, 2021 -- (Healthcare Sales & Marketing Network) -- Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant ... Biopharmaceuticals, FDA Protalix BioTherapeutics, Chiesi , Fabry disease, pegunigalsidase alfa (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 28, 2021 Category: Pharmaceuticals Source Type: news

Deerfield deal brings $65M to UB, Rochester drug development effort
The public-private partnership creates a new for-profit company to fund and support drug development projects for high-need therapeutic areas, including rare diseases. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 26, 2021 Category: Biotechnology Authors: Tracey Drury Source Type: news

An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future
A scientific vision for decades, gene therapy is finally becoming more common in the U.S., fueling optimism for the treatment of rare diseases. (Source: Reuters: Health)
Source: Reuters: Health - April 25, 2021 Category: Consumer Health News Source Type: news