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Rare Diseases & Genetics
Orphan conditions, registries, gene testing, and family support networks.
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Latest in Rare Diseases & Genetics
FDA, Duke-Margolis workshop to examine evidence standards for rare diseases (XBI:NYSEARCA)
20+ hour, 34+ min ago (114+ words) The “RISE to the Challenge: Statistical Considerations for Rare Disease Clinical Investigations” meeting is scheduled for Sept. 29 from 9 a.m. to 4:30 p.m. ET. The hybrid event will be held at the... The Food and Drug Administration and Duke-Margolis Institute for…...
18 months of unexplained symptoms led to my deadly diagnosis
3+ hour, 33+ min ago (1023+ words) By SHANIA OBRIEN, COMMERCIAL CONTENT PRODUCER (LIFESTYLE & SHOPPING) Taryn Elder was living what she calls her 'dream life', working in tourism marketing on the Gold Coast, exercising and enjoying Queensland's outdoors, when persistent health niggles quietly escalated. Recurring sinus infections…...
Battling rare disease, Jackson enjoys fire station visit while raising awareness
1+ day, 21+ hour ago (43+ words) Lake City Reporter A local youngster fighting a rare disease received a treat Thursday. In honor of National Kleefstra Syndrome Awareness Day, 7-year-old Eastside Elementary School student De’Mari Jackson received a tour…...
Why do some non-smokers get lung cancer? Rare gene offers new clues
2+ day, 5+ hour ago (712+ words) Lung cancer is usually linked to smoking, but some people who have never smoked also develop the disease. Scientists have been trying to understand why this happens and whether genes, pollution, workplace exposure, or other factors may increase the risk....
FDA Clears First Gene Therapy for Sanfilippo Syndrome Type A with Shipments to Treatment Centers Within 60 Days
2+ day, 3+ hour ago (783+ words) For the small number of American families living with this diagnosis, the approval turns an abstract hope into a series of concrete, urgent decisions. Parents now have to ask which hospitals will administer the infusion, whether their insurer will cover…...
Florida clinic is first in Southeast with gene therapy for fragile skin
2+ day, 5+ hour ago (475+ words) Approved treatment aids wound healing in children, adults with RDEB Written by Marisa Wexler, MS | Sept. 18, 2026 Zevaskyn (prademagene zamikeracel), a gene therapy approved in the U.S. last year to treat the fragile skin disease recessive dystrophic epidermolysis bullosa (RDEB), can now…...
Ultragenyx's $3.95 Million Fayuvi Secures First-Ever FDA Approval for Sanfilippo Syndrome Type A — BigGo Finance
2+ day, 1+ hour ago (792+ words) The U.S. Food and Drug Administration has cleared the first treatment ever for Sanfilippo syndrome Type A, awarding full approval to Ultragenyx Pharmaceutical Inc.'s (RARE) one-time gene therapy Fayuvi and handing the California drugmaker a valuable regulatory voucher in the…...
Ultragenyx Gene Therapy Is First Approved Treatment for Rare Neuro Disease Sanfilippo
1+ day, 23+ hour ago (408+ words) Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second approved gene therapy for Ultragenyx, which specializes in rare disease medicines. The trial failure…...
Abeona Therapeutics® Congratulates Ultragenyx on U.S. FDA Approval of FAYUVI™ Gene Therapy for Sanfilippo Syndrome Type A (MPS IIIA) | Taiwan News | Sep. 18, 2026 19:30
2+ day, 21+ hour ago (166+ words) - FDA approval represents a monumental milestone for patients with MPS IIIA and validates Abeona’s foundational R&D and clinical development efforts - - Ultragenyx successfully brought a life-changing therapy across the finish line for MPS IIIA patients and families - The approval of…...
Ultragenyx sets $3.95m list price for fatal rare disease therapy
2+ day, 8+ hour ago (400+ words) Ultragenyx Pharmaceuticals has set a US list price of $3.95m for its newly approved gene therapy, making it one of the world’s most expensive drugs. Fayuvi (rebisufligene etisparvovec-hopf) is a one-time treatment designed for children with a rare disease known as…...