Rare Diseases: Considerations for the Development of Drugs and Biological Products
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 22, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
$1M funding for AI drug discovery for rare diseases and more digital health fundings
Also, Cipla has increased its stake in digital pharma solutions company GoApptiv. (Source: mobihealthnews)
Source: mobihealthnews - December 22, 2023 Category: Information Technology Source Type: news
FABHALTA (iptacopan), FDA Approved for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria, Available at Biologics by McKesson
CARY, N.C., Dec. 20, 2023 — Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare diseases, was selected by Novartis as a specialty pharmacy provider for FABHALTA® (iptacopan). FABHALTA, approved by the U.S. Food and Drug Administration (FDA) on Dec. 5, 2023, is ... (Source: McKesson News)
Source: McKesson News - December 20, 2023 Category: Information Technology Source Type: news
FDA Approves Filsuvez (birch triterpenes) Topical Gel for the Treatment of Epidermolysis Bullosa
BOSTON, Dec. 19, 2023 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 19, 2023 Category: Drugs & Pharmacology Source Type: news
Topical Treatment for Epidermolysis Bullosa Wins FDA Approval
(MedPage Today) -- The FDA approved birch triterpenes (Filsuvez) topical gel for the treatment of epidermolysis bullosa (EB), Chiesi Global Rare Diseases announced on Tuesday.
The approval stipulates use for partial thickness wounds in adults... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - December 19, 2023 Category: American Health Source Type: news
Rare disease tech company was rolled up two years ago. Now it's spinning out again as an independent
This article originally appeared in the San Francisco Business Times, an American City Business Journals sister publication of Bizwomen.
Ciitizen, a Palo Alto, California company at the center of helping rare-disease patients collect vital medical records to jumpstart research into those conditions, is spinning out of genetic testing firm Invitae Corp.
San Francisco-based Invitae (NYSE: NVTA) bought Ciitizen only two years ago, hoping to tap the startup's expertise in collecting, organizing and … (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 14, 2023 Category: Biotechnology Authors: Ron Leuty Source Type: news
FDA Approves First CRISPR Treatment in U.S.
It was only 11 years ago that scientists Jennifer Doudna and Emmanuelle Charpentier first described a new way to edit genes, called CRISPR, in a scientific paper. The discovery is so game-changing that the pair earned the Nobel Prize in Chemistry in 2020 for how it could transform the way genetic diseases are treated. Now, on Dec. 8, the U.S. Food and Drug Administration (FDA) approved the very first treatment in the country based on the technology.
[time-brightcove not-tgx=”true”]
In the medical world, that’s lightning speed. “It’s incredible,” says Doudna, professor of chemistry a...
Source: TIME: Health - December 8, 2023 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized healthscienceclimate Source Type: news
RARE-X Launches Pompe Consortium to Support Patient-Owned Data Collection and Aggregated Data Sharing with Patient Community, Biopharma and Research Collaborators
Global Genes' research program, RARE-X, launches Pompe Consortium, gathering together individuals with rare diseases, advocacy organizations, biopharmaceutical companies, and academic researchers, to support patient-owned data collection and aggregated data sharing with patient community,... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - December 6, 2023 Category: Pharmaceuticals Tags: NPT PDT Source Type: news
OGSIVEO (nirogacestat) FDA Approved for Desmoid Tumors, Available at Biologics by McKesson
CARY, N.C., Dec. 5, 2023 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease, was selected by SpringWorks Therapeutics as a limited distribution specialty pharmacy for OGSIVEOTM (nirogacestat), which is a gamma secretase inhibitor indicated for adult pa... (Source: McKesson News)
Source: McKesson News - December 5, 2023 Category: Information Technology Source Type: news
TRUQAP (capivasertib), FDA Approved for the Treatment of Breast Cancer, Available at Biologics by McKesson
CARY, N.C., Dec. 1, 2023 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare diseases, was selected by AstraZeneca as a specialty pharmacy provider for TRUQAPTM (capivasertib). TRUQAP, which was approved by the U.S. Food and Drug Administration (FDA) on Nov. 17... (Source: McKesson News)
Source: McKesson News - December 1, 2023 Category: Information Technology Source Type: news
FDA Approves Ogsiveo (nirogacestat) for Adults with Desmoid Tumors
STAMFORD, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - November 27, 2023 Category: Drugs & Pharmacology Source Type: news
Medicines for four rare diseases become cheaper due to domestic production
Drugs for four of these diseases -Tyrosinemia Type 1, Gaucher's Disease, Wilson's Disease and Dravet-Lennox Gastaut Syndrome - along with sickle cell Anemia have been approved and are being manufactured indigenously. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - November 25, 2023 Category: Pharmaceuticals Source Type: news
Brave mother donates part of her liver to save son's life after he was diagnosed with a rare disease at just two years old
Catherine Baker, 42, from Sevenoaks, Kent, was heartbroken when her toddler son George was diagnosed with a rare condition. (Source: the Mail online | Health)
Source: the Mail online | Health - November 21, 2023 Category: Consumer Health News Source Type: news
S2 Episode 6: Rare Disease: Is It Membranous Nephropathy? S2 Episode 6: Rare Disease: Is It Membranous Nephropathy?
Join experts Drs Matthew Sparks and Laurence Beck as they discuss the diagnosis and management of membranous nephropathy, a rare kidney disease.Medscape (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - November 21, 2023 Category: Consumer Health News Tags: Nephrology InDiscussion Source Type: news
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
