Recognizing National Sickle Cell Awareness Month
September is National Sickle Cell Awareness Month and a time for us to underscore the biopharmaceutical industry ’s ongoing commitment to improve the lives of patients, families and communities affected by sickle cell disease. (Source: The Catalyst)
Source: The Catalyst - September 23, 2022 Category: Pharmaceuticals Authors: Anne McDonald Pritchett, PhD Tags: Medicines in Development Research and Development Rare Diseases Source Type: news

How Agile Clinical Trials Unlock Universal Access to Rare Disease...
In this free webinar, learn why the agile clinical trial model is a great fit for many rare disease categories. Attendees will learn how agile clinical trials are intrinsically patient-focused, how to...(PRWeb September 13, 2022)Read the full story at https://www.prweb.com/releases/how_agile_clinical_trials_unlock_universal_access_to_rare_disease_research_upcoming_webinar_hosted_by_xtalks/prweb18892963.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 13, 2022 Category: Pharmaceuticals Source Type: news

Centre could exclude low-cost meds, rare disease drugs from trade margin rationalisation
The confusion over which diseases are to be considered as “rare” and which drugs as “orphan” (used to treat rare diseases) has led to the decision of keeping them out of the ambit TMR as of now, people in the know told ET. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - September 7, 2022 Category: Pharmaceuticals Source Type: news

Political Squabbles Slow Drug Development for Rare Diseases
Political conflicts have stagnated the FDA's attempts to modernize clinical trials to effectively reflect the global population. Research for rare disease treatments lacks racial and ethnic...(PRWeb September 06, 2022)Read the full story at https://www.prweb.com/releases/political_squabbles_slow_drug_development_for_rare_diseases/prweb18879956.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 6, 2022 Category: Pharmaceuticals Source Type: news

CDER's ARC Program
The mission of CDER ’s ARC Program is to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - September 1, 2022 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Clinical trials: Japan ’s next chapter
Two years on from the start of the COVID-19 pandemic, and patients ’ lives have been upended. Many gave up visiting hospital even when they did not feel well. The pandemic and resulting lockdowns, terrible as they have been in so many ways, also served as a valuable testing ground for hybrid decentralized clinical trials (DCTs) in Japan. First, doctors performed medical examinations online for COVID-19 patients. Then, doctors or licensed nurses trained in good clinical practice (GCP) followed up on patients, visiting them at their homes or designated hotels.While online medical examinations during the pandemic have prov...
Source: EyeForPharma - August 31, 2022 Category: Pharmaceuticals Authors: Mary Nishikawa Source Type: news

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Brynn Duncan might as well live in a bubble. She suffers from mast cell activation syndrome, a rare disease that makes her allergic to almost everything. (Source: CNN.com - Health)
Source: CNN.com - Health - August 27, 2022 Category: Consumer Health News Source Type: news

HemoShear Therapeutics Appoints Brian Wamhoff, PhD, Interim Chief Executive Officer
Industry Veteran Robert Gould, PhD, Steps up to Board Chair CHARLOTTESVILLE, Va., Aug. 25, 2022 -- (Healthcare Sales & Marketing Network) -- HemoShear Therapeutics, Inc., a privately held biotechnology company developing drugs for rare diseases, today ... Biopharmaceuticals, Personnel HemoShear Therapeutics, REVEAL-Tx (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - August 25, 2022 Category: Pharmaceuticals Source Type: news

Global Monkeypox Cases Drop 21%, Reversing Month-Long Increase
GENEVA — The number of monkeypox cases reported globally dropped by 21% in the last week, reversing a month-long trend of rising infections and a possible signal the outbreak in Europe may be starting to decline, according to a World Health Organization report issued Thursday. The U.N. health agency reported 5,907 new weekly cases and said two countries, Iran and Indonesia, reported their first cases. To date, more than 45,000 cases have been reported in 98 countries since late April. Cases in the Americas accounted for 60% of cases in the past month, WHO said, while cases in Europe comprised about 38%. It said infec...
Source: TIME: Health - August 25, 2022 Category: Consumer Health News Authors: Associated Press Tags: Uncategorized Londontime monkeypox wire Source Type: news

Specialty Centers Treating Mesothelioma Lead Best Hospitals Rankings
Mesothelioma specialty programs played a significant role in determining the latest U.S. News & World Report Best Hospitals for Cancer Care annual rankings. For the eighth consecutive year, MD Anderson Cancer Center in Houston was ranked No. 1, followed by Memorial Sloan Kettering in New York; Mayo Clinic in Rochester, Minnesota; Dana Farber/Brigham and Women’s in Boston and UCLA Medical Center in Los Angeles. “This ranking is a reminder of our responsibility to those we serve, and it drives our unwavering commitment to our mission to end cancer,” said Dr. Peter WT Pisters, president of MD Anderson. “We a...
Source: Asbestos and Mesothelioma News - August 10, 2022 Category: Environmental Health Authors: Fran Mannino Tags: Doctors/Specialists Mesothelioma Treatment Source Type: news

Ashton Kutcher Reveals Rare Disease Diagnosis That Left Him Unable To See, Hear, Or Walk
Ashton Kutcher is opening up about an autoimmune disease he was diagnosed with that left him unable to see, hear, or walk. The actor shared the diagnosis in an upcoming episode of National Geograph… #nationalgeograph #ashtonkutcher (Source: Reuters: Health)
Source: Reuters: Health - August 9, 2022 Category: Consumer Health News Source Type: news

Ashton Kutcher says rare disease left him unable to see, hear, walk
Kutcher speaks about his experience with vasculitis on a coming episode of "Running Wild with Bear Grylls: The Challenge." #ashtonkutcher (Source: Reuters: Health)
Source: Reuters: Health - August 8, 2022 Category: Consumer Health News Source Type: news

54gene ’ s CEO Abasi Ene-Obong Wants to Fix the Racial Imbalance in Health Data
Born and raised in the Nigerian port city of Calabar, Abasi Ene-Obong remembers the exact moment that changed his life’s direction. Sitting in an introductory genetics class at medical school, in 2003, he heard the professor say that African genetic samples comprised less than 3% of health data bases in the world, creating a stunning vacuum in its ability to detect diseases and develop effective treatments for hundreds of millions of people. Ene-Obong ditched his plan to become a doctor, and instead left for London, and later Los Angeles, to study genetics, finally earning a Master’s degree in business focusing...
Source: TIME: Health - August 7, 2022 Category: Consumer Health News Authors: Vivienne Walt Tags: Uncategorized biztech2030 Innovation Leadership The Leadership Brief TIME 2030 Source Type: news

Bob Saget Scleroderma Fundraiser Turns Into Tribute Show, Celeb Pals Step In
Bob Saget's famous friends are stepping up to pick up the baton on Bob's fundraising for a rare disease. #bobsaget #baton #celebpalsstep (Source: Reuters: Health)
Source: Reuters: Health - August 3, 2022 Category: Consumer Health News Source Type: news

Study Suggests Relationship Between Stromal Tumors and Mesothelioma
Early identification of peritoneal mesothelioma cancer – when it is most effectively treated – may become more common after a recent study revealed a potential link to gastrointestinal stromal tumors. A single-center retrospective review by the division of surgical oncology at University of California San Diego Moores Cancer Center identified a previously underrecognized, synchronous co-occurrence between the two rare diseases. Annals of Surgical Oncology published the case series in July, which found that one in every 17 patients undergoing resection for gastrointestinal stromal tumors, known as GISTs, also had ...
Source: Asbestos and Mesothelioma News - August 2, 2022 Category: Environmental Health Authors: Fran Mannino Tags: Clinical Trials/Research/Emerging Treatments Peritoneal Source Type: news