All articles tagged with #rare disease
Originally Published 1 day ago — by CBS News
A man initially thought to have terminal lung cancer was finally diagnosed with a rare immune condition called IgG4-RD after a year of misdiagnosis, leading to a groundbreaking treatment trial that significantly improved his health and inspired advocacy efforts.
Originally Published 6 days ago — by USA Today
Jesy Nelson, former Little Mix member, shared that her twin daughters have been diagnosed with the severe and rare genetic disorder SMA Type 1, which may prevent them from walking or regaining neck strength. She emphasizes the importance of early treatment and raising awareness, expressing hope for her daughters' future despite the challenges.
Originally Published 20 days ago — by Shionogi Inc.
Shionogi plans to acquire global rights to Tanabe Pharma's RADICAVA (edaravone), a drug for ALS, to strengthen its presence in rare diseases and expand its rare disease portfolio, including establishing a strong U.S. commercial platform with a $2.5 billion investment.
Originally Published 21 days ago — by The Times
A mother undergoes a living donor liver transplant to save her daughter Ruby, who suffers from the rare metabolic condition PMM2-CDG, leading to significant health improvements and highlighting the emotional and medical complexities of such procedures.
Originally Published 1 month ago — by TelegraphHerald.com
A man from Dubuque is learning to navigate life with a rare disease, sharing his ongoing journey and challenges faced along the way.
Originally Published 2 months ago — by PennLive.com
Young actress Isabelle Tate, known for her role in '9-1-1 Nashville,' passed away at 23 due to a rare form of Charcot-Marie-Tooth disease, the same chronic neuromuscular condition that country music star Alan Jackson is battling, highlighting the impact of this progressive disease on individuals' lives.
Originally Published 2 months ago — by BuzzFeed
The article shares a mother's emotional journey and lessons learned after her daughter's diagnosis with a rare, incurable autoimmune disease, emphasizing the importance of sitting with pain, growth through adversity, and redefining normal life after trauma.
Originally Published 2 months ago — by PennLive.com
Young actress Isabelle Tate, known for her role in '9-1-1 Nashville,' died at 23 from a rare form of Charcot-Marie-Tooth disease, the same chronic neuromuscular condition that country music icon Alan Jackson is battling, highlighting the impact of this progressive disease on young lives.
Originally Published 3 months ago — by Yahoo
A young boy named Jack, born prematurely and initially healthy, was diagnosed with a rare, terminal genetic disorder called INAD, which leads to progressive loss of motor and cognitive functions. Despite no current cure, a promising gene therapy is in development, and a community-funded effort is underway to fund clinical trials that could extend Jack's life and improve his quality of life. The family remains hopeful and focused on making the most of their time together.
Originally Published 3 months ago — by BBC
A cluster of young adults in south Wales is affected by DRPLA, a rare inherited neurological disorder with no current cure, prompting efforts to raise awareness, improve diagnosis, and explore potential treatments through clinical trials.
Originally Published 4 months ago — by PennLive.com
Fox News anchor John Roberts, age 70, is battling a rare and severe case of malaria, a disease caused by a mosquito-borne parasite, which is unusual in the United States. He shared his health update on social media, and fans are sending well-wishes for his recovery.
Originally Published 4 months ago — by BBC
A father from Bath is raising awareness and funds for research into Phelan-McDermid Syndrome, a rare genetic condition affecting only about 300 people worldwide, in hopes of finding a cure or effective therapies for his son Jenson and others with the condition.
Originally Published 4 months ago — by Salina Post
A man infected with the deadly brain-eating amoeba Naegleria fowleri has died in Missouri, possibly linked to water activities at Lake of the Ozarks. The infection, extremely rare and not contagious between people, occurs when contaminated water enters the nose, traveling to the brain. Public health officials are investigating the source, and precautions such as avoiding submerging the head in warm freshwater are recommended to reduce risk.
Originally Published 4 months ago — by NBC News
Parents of children with Barth syndrome are urgently seeking FDA approval for the experimental drug elamipretide, which has shown promise in treating the rare disease but faces delays due to manufacturing issues and regulatory hurdles, risking the lives of affected children.
Originally Published 4 months ago — by statnews.com
Stealth BioTherapeutics disclosed an FDA rejection letter to rally support for its experimental drug for Barth syndrome, a rare disease affecting about 150 people in the U.S., amid ongoing struggles to meet regulatory requirements and concerns over the company's future.