FDA Grants Orphan Drug Designation to NeoImmuneTech's NT-I7 for the Treatment of Acute Radiation Syndrome
The FDA's Orphan Drug Designation is aimed at advancing treatments for rare diseases, including Acute Radiation Syndrome (ARS), which currently has limited treatment options
NeoImmuneTech's NT-I7 has shown potential in nonclinical studies to address the... Biopharmaceuticals, FDA NeoImmuneTech, efineptakin alfa, Acute Radiation Syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 20, 2023 Category: Pharmaceuticals Source Type: news
FRUZAQLA (fruquintinib), FDA Approved for the Treatment of Metastatic Colorectal Cancer, Available at Biologics by McKesson
CARY, N.C., Nov. 15, 2023 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare diseases, was selected by Takeda as a specialty pharmacy provider for FRUZAQLATM (fruquintinib). FRUZAQLA, approved by the U.S. Food and Drug Administration (FDA) on Nov. 8, 2023, is ... (Source: McKesson News)
Source: McKesson News - November 15, 2023 Category: Information Technology Source Type: news
AstraZeneca signs deal for oral obesity drug and lifts profit outlook
Anglo-Swedish drugmaker’s oncology and rare disease medication sales offset waning demand for Covid jabs (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - November 9, 2023 Category: Pharmaceuticals Source Type: news
Family's gene therapy startup wins historic OK for clinical trial targeting daughter's rare disease
Less than 100 people have been identified with life-threatening NGLY1 deficiency. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - November 8, 2023 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
Government plans to offer support for treatment of rare diseases
"Accordingly, as these patients have already commenced treatment, Sarepta Therapeutics has committed, as per the communication received by the NDRC (National Rare Diseases' Committee), to continue treating these patients with Duchenne muscular dystrophy for the next three months, and to supply the medication free of cost during this period," it said. The court directed doctor Madhulika Kabra from the All India Institute of Medical Science to place the order for the medicine with the company within one week and expedite the procurement for these 14 patients. (Source: The Economic Times)
Source: The Economic Times - November 7, 2023 Category: Consumer Health News Source Type: news
First Treatment in 30 Years for Rare Disease Safe, Effective First Treatment in 30 Years for Rare Disease Safe, Effective
Subcutaneous efgartigimod PH20 SC is effective and tolerable for the treatment of CIDP, providing a convenient alternative to intravenous and other older treatments.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 6, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Indo US Bridging Rare Summit Heralds a New Era of Cooperation for Rare Diseases Orphan Drugs Development
The FDA's Commissioner, Dr. Robert M. Califf, recently visited India and shared his opinion on India's Unique Opportunity and Important Responsibility as the Pharmacy to the World. The Inaugural 2-day Indo US Bridging RARE Summit was held on Oct 29 and 30, 2023 at George Mason University... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 6, 2023 Category: Pharmaceuticals Tags: NPT Source Type: news
CDER Continues to Advance Rare Disease Drug Development with New Efforts, Including the Accelerating Rare Disease Cures (ARC) Program
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - November 2, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
What to Know About Hypertrophic Cardiomyopathy in Kids
The human heart is a muscle, but it’s also a kind of complicated balloon—a balloon that fills and empties roughly 60 to 100 times every minute, and several billion times during the course of a lifetime.
Among people with hypertrophic cardiomyopathy, the walls of the heart muscle are abnormally thick. This thickness can interfere with the heart’s normal filling-and-emptying operation. “If you think of a balloon made with super-thick rubber, you have to blow harder to fill it, and it’s the same with a hypertrophic heart,” says Dr. Daphne Hsu, professor of pediatrics and medicine at Ped...
Source: TIME: Health - November 2, 2023 Category: Consumer Health News Authors: Markham Heid Tags: Uncategorized freelance healthscienceclimate Source Type: news
Govt set to file progress report on talks with makers of rare disease drugs
Activists have demanded that tax exemption be given for such drugs that are approved by India’s drug regulator and are available in the country. During the last hearing, the government counsel informed the court it has flagged the issue over customs duty and GST to the finance ministry and “is hopeful of informing the court about the outcome of the said issue on the next date”. (Source: The Economic Times)
Source: The Economic Times - November 1, 2023 Category: Consumer Health News Source Type: news
Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement - 12/14/2023
On December 14th, 2023, FDA and Duke Margolis will host a public meeting to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence to support rare disease drug approvals. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - November 1, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
AstraZeneca announces collaboration and investment agreement with Cellectis to accelerate cell therapy and genomic medicine ambitions
AstraZeneca today announced a collaboration and investment agreement with Cellectis, a clinical-stage biotechnology company, to accelerate the development of next generation therapeutics in areas of high unmet need, including oncology, immunology and rare diseases. (Source: World Pharma News)
Source: World Pharma News - November 1, 2023 Category: Pharmaceuticals Tags: Featured AstraZeneca Business and Industry Source Type: news
November 2023
Be a Citizen Scientist : Help Researchers Solve Puzzling Problems
Insights Into Long COVID
Sore, Scratchy Throat? : Soothing Pain From Illness or Allergy
Helping a Child Who Is Overweight
Genetic and Rare Diseases Information Center (Source: NIH News in Health)
Source: NIH News in Health - October 31, 2023 Category: Consumer Health News Source Type: news
His Rare Disease's Cure Was Sitting on the Pharmacy Shelf His Rare Disease's Cure Was Sitting on the Pharmacy Shelf
He ' s alive thanks to a repurposed drug. Now his dream is to find cures for millions of people from among drugs we already have.Medscape (Source: Medscape Allergy Headlines)
Source: Medscape Allergy Headlines - October 31, 2023 Category: Allergy & Immunology Tags: Internal Medicine Expert Interview Source Type: news
X4 Pharmaceuticals Announces FDA Acceptance with Priority Review of U.S. NDA for Mavorixafor in WHIM Syndrome
BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) --X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the United States Food and Drug Administration (FDA) has... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - October 31, 2023 Category: Drugs & Pharmacology Source Type: news
