Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars
Rare diseases have gained more attention in recent years, perhaps in part due to the high price tags often associated with approved therapies. But the cost of rare disease drugs is dwarfed by the cost of not having them. Our recent study shows that a lack of treatment for a rare disease is…#orphandrugtaxcredit #fda #petermarks #arc #learningandeducation #cder #nih #als #giacomochiesi #chiesigroup (Source: Reuters: Health)
Source: Reuters: Health - May 25, 2023 Category: Consumer Health News Source Type: news
Little girl's rare disease highlights new opportunities — and challenges — in gene therapy
In a followup to our story last summer, an East Bay family is using the Ice Bucket Challenge model to raise money to develop and manufacturing a gene therapy for a clinical trial for their 2-year-old daughter with a rare genetic deficiency. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 25, 2023 Category: American Health Authors: Ron Leuty Source Type: news
Setting the record straight on accelerated approval
As we ’ve discussed on thisblogbefore, the U.S. Food and Drug Administration ’s (FDA)accelerated approval program has served as a critical lifeline for patients with serious and life-threatening conditions. The program was originally established at the urging of patient advocates during the height of the HIV/AIDS epidemic, in which patients waited years for the FDA to review and approve new treatments. From its inception three decades ago, the pathway ’s purpose has always been to get medicines to patients most in need, especially those who lack any viable treatment options. Without accelerated approval, patients in ...
Source: The Catalyst - May 22, 2023 Category: Pharmaceuticals Tags: FDA Rare Diseases Accelerated Approval Source Type: news
Insilico Medicine delivers preclinical candidate targeting ENPP1 for cancer immunotherapy and the treatment of rare disease using generative AI
Insilico Medicine (Insilico), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, announced that the company has nominated a potentially best-in-class preclinical candidate targeting ENPP1 for cancer immunotherapy and the potential treatment of Hypophosphatasia (HPP).
ENPP1 is an ecto-nucleotide pyrophosphatase that plays an important role in purinergic signaling that regulates immune, cardiovascular, neurological, and hematological system functions. (Source: World Pharma News)
Source: World Pharma News - May 22, 2023 Category: Pharmaceuticals Tags: Featured Business Business and Industry Source Type: news
Rare Disease Families Advocate For Change
Kasey and Doug Woleben joined other families to establish the all-volunteer Cure Mito Foundation to help find a cure or treatment for Leigh syndrome. (Source: WebMD Health)
Source: WebMD Health - May 19, 2023 Category: Consumer Health News Source Type: news
Maine Confirms Fatal Powassan Virus Case. What to Know About the Rare Disease
A Maine resident has died from the Powassan virus, a rare tick-borne illness, Maine’s Centers for Disease Control confirmed on Wednesday.
The individual, who was likely infected in the state, developed neurologic symptoms and died while in the hospital, according to Maine’s CDC.
This is the state’s first case of Powassan virus this year. In 2022, two individuals died from the illness, making this the third recorded Powassan death in Maine since 2015.
Read More: Ticks Carry More Diseases Than Just Lyme. Here’s What You Need to Know
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Like Lyme disease, whi...
Source: TIME: Health - May 19, 2023 Category: Consumer Health News Authors: Simmone Shah Tags: Uncategorized General Assignment Public Health Source Type: news
FDA 'Leans In' to Accelerated Approval for Rare Disease Drugs
(MedPage Today) -- The FDA is "leaning in" to cell and gene therapies, expressing a willingness to use the accelerated approval pathway for these products, particularly as new leadership is set to take charge of a recently revamped office.
That... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - May 19, 2023 Category: American Health Source Type: news
FDA Approves Vyjuvek (beremagene geperpavec-svdt) Redosable Gene Therapy for the Treatment of Dystrophic Epidermolysis Bullosa
PITTSBURGH, May 19, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the Company) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, today announced the US Food and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 19, 2023 Category: Drugs & Pharmacology Source Type: news
Janssen to Highlight Scientific Advances and Commitment to Transform Cancer Care at ASCO and EHA with More than 90 Presentations Showcasing Robust, Differentiated Portfolio and Pipeline in Hematologic Malignancies and Solid Tumors
This study evaluates an investigational combination regimen of IMBRUVICA® (ibrutinib) plus venetoclax (I+V) for newly diagnosed chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), (Abstracts #7535 and #P617, respectively).Advancing the Science of Solid Tumors Through Precision MedicineJanssen’s continued innovation in solid tumors focuses on advancing precision medicine options for patients with biomarker-driven disease who have limited targeted treatment options and moving patients into earlier lines of therapy when treatments may be more effective. Lung CancerAn oral presentation will report ...
Source: Johnson and Johnson - May 18, 2023 Category: Pharmaceuticals Tags: Innovation Source Type: news
NIH foundation, partnering with research institutions, adds 8 rare diseases to clinical trial portfolio
The Foundation for the National Institutes of Health has added eight rare diseases to its gene therapy-focused clinical trial portfolio, betting that a public-private partnership can grease an oft- | The Foundation for the National Institutes has tacked on eight new rare diseases to its Bespoke… (Source: Reuters: Health)
Source: Reuters: Health - May 17, 2023 Category: Consumer Health News Source Type: news
The Foundation for the National Institutes of Health Announces Selection of Eight Rare Diseases for the Bespoke Gene Therapy Consortium Clinical Trial Portfolio
NORTH BETHESDA, MD, May 16, 2023 – Today, the Foundation for the National Institutes of Health (FNIH) announces the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) has selected eight rare diseases for its clinical trial portfolio. This portfolio pioneers a novel…#northbethesda #fnih #nih #fda #bgtc #juliegerberding #adeno #aav #terrypirovolakis #elpidatherapeutics (Source: Reuters: Health)
Source: Reuters: Health - May 17, 2023 Category: Consumer Health News Source Type: news
FTC Suing To Stop Amgen ’s $27.8 Billion Purchase Of Horizon Therapeutics
The FTC is attempting to stop the sale out of fears it will give the company too much power over rare disease treatments. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - May 16, 2023 Category: Pharmaceuticals Authors: Molly Bohannon, Forbes Staff Tags: Business /business Innovation /innovation Healthcare /healthcare Breaking breaking-news topline Source Type: news
'I've been stuck in my bedroom for two years'
A young woman with a rare disease says she is not being supported to have any quality of life. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - May 16, 2023 Category: Consumer Health News Source Type: news
When does life end? New organ donation strategy fuels debate
On a chilly holiday Monday in January 2020, a medical milestone passed largely unnoticed. In a New York City operating room, surgeons gently removed the heart from a 43-year-old man who had died and shuttled it steps away to a patient in desperate need of a new one.
More than 3500 people in the United States receive a new heart each year. But this case was different—the first of its kind in the country. “It took us 6 months to prepare,” says Nader Moazami, surgical head of heart transplantation at New York University (NYU) Langone Health, where the operation took place. The run-up included oversight from an eth...
Source: ScienceNOW - May 11, 2023 Category: Science Source Type: news
FDA Approves Elfabrio (pegunigalsidase alfa-iwxj) for the Treatment of Adult Patients with Fabry Disease
BOSTON and CARMIEL, Israel, May 10, 2023 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics,... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 10, 2023 Category: Drugs & Pharmacology Source Type: news
