FDA awards 11 grants to clinical trials to develop new medical products for rare disease treatments
Today, the U.S. Food and Drug Administration announced it has awarded 11 new clinical trial research grants, equaling more than $25 million of funding over the next four years. The FDA's Congressionally-funded Orphan Products Grants Program awards these grants to clinical investigators to support the development of medical products for patients with rare diseases. (Source: World Pharma News)
Source: World Pharma News - October 14, 2021 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

Peninsula drug company regroups after failed study in deadly rare disease in kids
A drug from a small, ambitious Peninsula drug developer failed a clinical trial for an invariably fatal childhood genetic disease, but leaders of the company said they will regroup with the FDA to find a way to move the program forward. Retrotope Inc. said the late-stage study of its drug RT-001 failed to hit its main goal in infantile neuroaxonal dystrophy, or INAD, a rare condition which robs children of motor skills and voluntary muscle control and typically kills them before they turn 10. But… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - October 7, 2021 Category: Health Management Authors: Ron Leuty Source Type: news

Peninsula drug company regroups after failed study in deadly rare disease in kids
A drug from a small, ambitious Peninsula drug developer failed a clinical trial for an invariably fatal childhood genetic disease, but leaders of the company said they will regroup with the FDA to find a way to move the program forward. Retrotope Inc. said the late-stage study of its drug RT-001 failed to hit its main goal in infantile neuroaxonal dystrophy, or INAD, a rare condition which robs children of motor skills and voluntary muscle control and typically kills them before they turn 10. But… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 7, 2021 Category: Biotechnology Authors: Ron Leuty Source Type: news

Virtual Symposium to Focus on Mesothelioma Treatment Advances
Sylvester Comprehensive Cancer Center of South Florida will host its free virtual First Miami International Mesothelioma Symposium on November 6, 2021. The Mesothelioma Program at the Sylvester Comprehensive Cancer Center has grown to become one of America’s leaders in this specialized field, attracting a wide range of expertise for its upcoming virtual symposium. “Mesothelioma is such a rare disease that we really have to bring in experts from around the world to get a true picture of what’s coming next. And I think we’ve done that with our program,” medical oncologist Dr. Estelamari Ro...
Source: Asbestos and Mesothelioma News - October 5, 2021 Category: Environmental Health Authors: Amy Edel Source Type: news

Three research fellows earn opportunity to grow in their careers
Supporting scientists in Africa, understanding a rare disease, and better communicating study findings are among the NIEHS trainees’ goals. (read more) (Source: Environmental Factor - NIEHS Newsletter)
Source: Environmental Factor - NIEHS Newsletter - October 2, 2021 Category: Environmental Health Source Type: news

Peninsula biotech looks to turn once-failed osteoporosis drug against rare disease
A Bay Area company went on the hunt for a certain class of drugs that could level off calcium levels in both the blood and urine. Now it plans to take a drug into a late-stage clinical trial for people at risk of deadly kidney disease. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 1, 2021 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Peninsula biotech looks to turn once-failed osteoporosis drug against rare disease
A Bay Area company went on the hunt for a certain class of drugs that could level off calcium levels in both the blood and urine. Now it plans to take a drug into a late-stage clinical trial for people at risk of deadly kidney disease. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 1, 2021 Category: Biotechnology Authors: Ron Leuty Source Type: news

Peninsula biotech looks to turn once-failed osteoporosis drug against rare disease
A Bay Area company went on the hunt for a certain class of drugs that could level off calcium levels in both the blood and urine. Now it plans to take a drug into a late-stage clinical trial for people at risk of deadly kidney disease. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 1, 2021 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Peninsula biotech looks to turn once-failed osteoporosis drug against rare disease
A Bay Area company went on the hunt for a certain class of drugs that could level off calcium levels in both the blood and urine. Now it plans to take a drug into a late-stage clinical trial for people at risk of deadly kidney disease. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 1, 2021 Category: Biotechnology Authors: Ron Leuty Source Type: news

Castle Creek to collaborate with Mayo Clinic on developing new gene therapies for rare diseases
Castle Creek Biosciences Inc. is teaming up with the Mayo Clinic to advance the development of potential gene therapies for two rare genetic connective tissue disorders. "We expect this initiative will be the first of multiple Castle Creek strategic collaborations with leading medical research institutions that have the potential to expand our innovative gene therapy discoveries for rare diseases," said Matthew Gantz, CEO of Castle Creek Biosciences. The partners hip will focus on the discovery… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 30, 2021 Category: Pharmaceuticals Authors: John George Source Type: news

Study Shows Access to Care Often Difficult for Mesothelioma Patients
When it comes to malignant pleural mesothelioma, not everyone is treated equally. A disparity in access to care makes the difference in mesothelioma treatment quite dramatic, according to the University of Miami’s Sylvester Comprehensive Cancer Center. “Patients without access to expert multidisciplinary care at high-volume, academic facilities are being inappropriately undertreated for their disease,” medical oncologist Dr. Estelamari Rodriguez told The Mesothelioma Center at Asbestos.com. “The access is not adequate as it should be for everyone.” Rodriguez led a recent study at Univers...
Source: Asbestos and Mesothelioma News - September 28, 2021 Category: Environmental Health Authors: Chris Elkins Source Type: news

What Looks Like Acrocyanosis?
Discussion Acrocyanosis is a peripheral vascular disease where the skin and mucous membranes have a persistent, painless, deep-bluish color due to decreased oxygenated blood which usually is benign. The deoxygenated blood can be due to a central cause such as cyanotic congenital heart disease or localized problems often due to vasospasm. Hands and feet are the most common areas affected. It is thought that there is “…vasospasm of the cutaneous arteries, and arterioles along with compensatory dilatation in the capillary and post capillary venules caus[ing] cyanosis and sweating.” Palms and soles may or ma...
Source: PediatricEducation.org - September 27, 2021 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders
Basel, 24 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investig ation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).“These new data for Evrysdi may help extend the potential ben...
Source: Roche Media News - September 24, 2021 Category: Pharmaceuticals Source Type: news

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders
Basel, 24 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investig ation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).“These new data for Evrysdi may help extend the potential ben...
Source: Roche Investor Update - September 24, 2021 Category: Pharmaceuticals Source Type: news

Genomenon Announces Collaboration to Accelerate Genetic Diagnosis for...
Genomenon to Make Essential Genomic Data for Rare Diseases Available to Genetic Testing Labs Worldwide(PRWeb September 20, 2021)Read the full story at https://www.prweb.com/releases/genomenon_announces_collaboration_to_accelerate_genetic_diagnosis_for_rare_disease_patients/prweb18204712.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 20, 2021 Category: Pharmaceuticals Source Type: news

Latest-analyses-Vitrakvi-larotrectinib-reaffirm-powerful-efficacy-tolerability-profile-adult
In an updated analysis on the adult subset of non-central nervous system (CNS) / TRK fusion cancer patients from three larotrectinib clinical trials, a high overall response rate (ORR) of 67% with a median duration of response (mDoR) of 49.3 months was observed as well as an extended progression-free survival (PFS; median of 25.8 months) as assessed by investigators / Post hoc sub-analysis of investigator-assessed ORR, DoR and PFS of patients (n=218) show uniformly high ORRs in patients regardless of prior treatment or performance status; highest response rates observed in patients receiving first-line larotrectinib (81%) ...
Source: Bayer Company News - September 16, 2021 Category: Pharmaceuticals Source Type: news

This Startup Just Raised $50 Million To Deliver Gene Therapies For Rare Diseases
iECURE aims to use gene editing to treat patients suffering from rare genetic disorders. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - September 9, 2021 Category: Pharmaceuticals Authors: Aayushi Pratap, Forbes Staff Tags: Healthcare /healthcare Innovation /innovation Editors' Pick editors-pick Source Type: news

How does Lichen Sclerosus et Atrophicus Present?
Discussion Lichen sclerosus et atrophicus (LSA) is a chronic inflammatory disease with a strong autoimmune association. It can occur with other autoimmune diseases. Its specific cause is unknown. It affects females and males of all ages, but especially females 40-60 years. In the pediatric population it affects prepubertal females most often. Treatment includes steroid medications or anti-inflammatory medications. Other treatments may be offered including phototherapy, and much less commonly systemic steroids or immunosuppressive therapy. Circumcision may also be an option for some males. Childhood LSA generally improves b...
Source: PediatricEducation.org - September 6, 2021 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

Tanzania: Zanzibar Minister Lauds Genetics Database Plan
[Citizen] Dar es Salaam -- The Zanzibar minister for Health, Mr Nassor Mazrui, has commended the Tanzania Society of Human Genetics (TSHG) plan of establishing a national genetic database, saying it will be crucial in providing scientists with relevant data for rare disease diagnostic. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - September 4, 2021 Category: African Health Source Type: news

Prime Therapeutics rises to Minnesota's No. 2 largest private company as pharmacy costs rise
CEO Ken Paulus said the company ’s steady growth has largely been driven by increases in pharmacy spending, specifically for prescription medications used to treat complex conditions including oncology, autoimmune and rare diseases. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - September 2, 2021 Category: American Health Authors: Carter Jones Source Type: news

Prime Therapeutics rises to Minnesota's No. 2 largest private company as pharmacy costs rise
CEO Ken Paulus said the company ’s steady growth has largely been driven by increases in pharmacy spending, specifically for prescription medications used to treat complex conditions including oncology, autoimmune and rare diseases. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 2, 2021 Category: Biotechnology Authors: Carter Jones Source Type: news

Biopharmaceuticals: Sobi deal could reignite Pfizer and Biogen interest
More bids for the rare disease drugmaker are likely to come to the table (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - September 2, 2021 Category: Pharmaceuticals Source Type: news

Rare disease company EnBiotix merges with Swiss firm
EnBiotix Inc., a Boston-based biotech focused on rare diseases, is merging with Swiss oncology and antimicrobial resistance-focused company Polyphor. Under the terms of the agreement, Polyphor will acquire all of EnBiotix's outstanding capital stock; EnBiotix will in turn acquire the rights to inhaled murepavadin, a potent antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in cystic fibrosis patients, for $10 million . The merger is expected to close in the fourth quarter… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 1, 2021 Category: Pharmaceuticals Authors: Rowan Walrath Source Type: news

Rare disease company EnBiotix merges with Swiss firm
EnBiotix Inc., a Boston-based biotech focused on rare diseases, is merging with Swiss oncology and antimicrobial resistance-focused company Polyphor. Under the terms of the agreement, Polyphor will acquire all of EnBiotix's outstanding capital stock; EnBiotix will in turn acquire the rights to inhaled murepavadin, a potent antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in cystic fibrosis patients, for $10 million . The merger is expected to close in the fourth quarter… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 1, 2021 Category: Biotechnology Authors: Rowan Walrath Source Type: news

Yale geneticist with rare disease to build pediatric muscle cell atlas
A $3 million grant will support research led by YSM ’s Monkol Lek to create a roadmap of healthy skeletal cells and how they change at four key age milestones. (Source: Yale Science and Health News)
Source: Yale Science and Health News - August 31, 2021 Category: Universities & Medical Training Source Type: news

Discharged Snowboarder Dies of Internal Bleeding; Hospital Sued Discharged Snowboarder Dies of Internal Bleeding; Hospital Sued
A hospital discharges an injured snowboarder who then dies of internal bleeding; a toddler dies when his rare disease is not diagnosed; actions against docs who lie about their vaccination status.Medscape Business of Medicine (Source: Medscape Business of Medicine Headlines)
Source: Medscape Business of Medicine Headlines - August 31, 2021 Category: Pharmaceuticals Tags: Family Medicine/Primary Care News Source Type: news

Study: Pleural Mesothelioma Treatment Effective with Peritoneal Disease
We report the first real-world evidence regarding clinical outcomes for a cohort of patients with advanced MPeM receiving ICIs,” the MD Anderson study authors wrote. “Our results provide much-needed data supporting the role of ICIs in patients with this rare disease, who cannot participate in clinical trials and otherwise have no or limited treatment options.” The post Study: Pleural Mesothelioma Treatment Effective with Peritoneal Disease appeared first on Mesothelioma Center - Vital Services for Cancer Patients & Families. (Source: Asbestos and Mesothelioma News)
Source: Asbestos and Mesothelioma News - August 31, 2021 Category: Environmental Health Authors: Fran Mannino Source Type: news

Why this tech company raised $50M to tackle dozens of rare diseases
"Drug companies don't have a lot of great options when it comes to getting the data and how to design clinical trials," the company's CEO said. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - August 24, 2021 Category: Health Management Authors: Ron Leuty Source Type: news

AllStripes lands $50M for tool that helps patients share medical data
The platform lets patients with rare diseases access their medical data and participate in research studies. (Source: mobihealthnews)
Source: mobihealthnews - August 24, 2021 Category: Information Technology Source Type: news

Pharma companies' charity hope for kids with rare spinal disorder
The rare disease affects one in every 8,000 children. The bigger problem, according to parents and doctors, is the lack of awareness about rare diseases. By the time parents reach out to the doctors, there has already been a delay. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - August 22, 2021 Category: Pharmaceuticals Source Type: news

WELIREGTM (belzutifan), FDA Approved, Available at Biologics by McKesson
CARY, N.C., August 20, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Merck& Co. as a specialty pharmacy provider for WELIREGTM (belzutifan), an inhibitor of hypoxia-inducible factor-2a (HIF-2a) indicated for the t... (Source: McKesson News)
Source: McKesson News - August 20, 2021 Category: Information Technology Source Type: news

Alex Gorsky to Serve as Executive Chairman and Transition Role of Chief Executive Officer of Johnson & Johnson to Joaquin Duato, Effective January 3, 2022
New Brunswick, N.J. (August 19, 2021) – Johnson & Johnson (NYSE: JNJ) today announced that Mr. Alex Gorsky, currently Chairman and Chief Executive Officer, will serve as Executive Chairman of Johnson & Johnson and transition the Chief Executive Officer role to Mr. Joaquin Duato, currently Vice Chairman of the Company’s Executive Committee, effective January 3, 2022. Following the transition of the Chief Executive Officer role, Mr. Duato will also be appointed as a member of the Company’s Board of Directors. “It has been an honor and privilege to lead this company as Chairman and CEO for near...
Source: Johnson and Johnson - August 19, 2021 Category: Pharmaceuticals Tags: Our Company Source Type: news

The Petri Dish: Chaos at Sesen Bio, funding for rare disease drugs
While some in the life sciences industry create drugs and devices to tackle the Covid-19 pandemic, their colleagues are busy as ever raising money, testing new drugs, expanding facilities and more. Here's The Petri Dish, a roundup of news that may get overlooked amid the influx of Greater Boston life sciences and health care happenings. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 19, 2021 Category: Pharmaceuticals Authors: Rowan Walrath Source Type: news

ClinOne Revenue Soars 165% in First Half of 2021 as Pharma Speeds...
First half growth, including 20 new customers, driven primarily in complex therapeutic areas including oncology, rare disease, CNS, cardiovascular, and NASH(PRWeb August 11, 2021)Read the full story at https://www.prweb.com/releases/clinone_revenue_soars_165_in_first_half_of_2021_as_pharma_speeds_adoption_of_technology_for_peer_referrals_econsent_and_patient_empowerment_in_global_clinical_research/prweb18126411.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - August 11, 2021 Category: Pharmaceuticals Source Type: news

Fueling innovation: What following the science has taught one biotech
The bar for innovation has never been higher. Regardless of the industry, today ’s challenges require an unparalleled level of ingenuity and a diversity of skillsets and viewpoints that can be achieved only through collaboration. To build something truly meaningful today, organizations must surround themselves with visionaries who bring different talents to the table while be ing united in a common goal. Take the pursuit of treatments for rare disease. According to the National Institutes of Health,… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - August 2, 2021 Category: American Health Authors: Sharon Barr, Ph.D., Senior Vice President, Head of Research and Product Development and Gianluca Pirozzi, M.D., Senior Vice President, Head of Development and Safety Source Type: news

DisperSol and Catalent Collaborate to Establish KinetiSol ® ...
DisperSol Technologies, a clinical-stage pharmaceutical company developing new treatments for oncology and rare diseases, and Catalent, the leading global provider of advanced delivery technologies,...(PRWeb August 02, 2021)Read the full story at https://www.prweb.com/releases/dispersol_and_catalent_collaborate_to_establish_kinetisol_technology_manufacturing_hub_for_dispersol_pharmaceutical_pipeline/prweb18106760.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - August 2, 2021 Category: Pharmaceuticals Source Type: news

Texas Monkeypox Case Underscores Need for Better Surveillance
A patient caught the rare disease in Nigeria before flying through two US airports, exposing more than 200 people from 27 states. (Source: The Scientist)
Source: The Scientist - July 30, 2021 Category: Science Tags: News & Opinion Source Type: news

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