Cystic Fibrosis & Multiple Sclerosis, Muscular Dystrophy updates

[waltky]

Sad story of a young couple with cystic fibrosis...

Terminally ill US couple die days apart
Thu, 22 Sep 2016 - A young married couple who bonded through their shared struggles with cystic fibrosis have both died within days of each other.

A young US married couple who bonded through their shared struggles with cystic fibrosis have both died within days of each other. Katie Prager, 26, died in Kentucky on Thursday, five days after the death of her husband, Dalton Prager, 25. Katie died in bed on Thursday morning, her mother said wrote on Facebook. "I know Dalton was waiting with open arms, as well as both her grandmothers and a host of family and friends that have gone before her," she wrote. The couple married in 2011 after falling in love on Facebook. Doctors had advised against meeting in person, due to the likelihood that Dalton would pass a highly-infectious disease to Katie.

The young couple on a beach

The couple had been forced to live in separate states in recent years and were last together for their fifth anniversary in July. Katie had lived in Kentucky, while Dalton was living in Missouri. Cystic fibrosis clogs the lungs with mucus making it difficult for patients to breath. The median survival age is 40-years-old. They had each received lung transplants, which failed. Dalton had tried desperately but was unable to travel to Kentucky to see Katie one last time before his death. "He has tried so many times and he has tried so hard. Unfortunately his body is not agreeing with what he wants to do," his mother Renee Prager told the St Louis Post-Dispatch. The couple raised money for their treatment, and wrote extensively about their difficult experiences.

Lamenting how he and his wife were treated by health insurance companies, Dalton wrote: "They are turning my wife into a number, a statistic, a dollar sign. I cannot lose her! This can't be the end of our love story! "We are both ready to continue fighting but at this point we are running out of options and need your help. Please help me save my wife Katie! "We knew her time was short and she was able to do a few things that she wanted," Katie's mother Debbie wrote. "The days to follow will not be easy but I find comfort in knowing that my girl lived, she really lived." The couple were compared to the one in the book and film The Fault In Our Stars, that focuses on a young couple battling cancer.

http://www.bbc.co.uk/news/world-us-canada-37442522

[The Xl]

This hits me hard as my brother has cystic fibrosis. Rip to them both.

[valley ranch]

This is a terrible thing, this disease, we've helped several to enjoy skiing. They're so perfect otherwise, and love life and appreciate everything.

[waltky]

Wonder if it'll work for Cystic Fibrosis?...

Shark-inspired drug may help treat fibrosis, researchers say
Mon, 30 Jan 2017 - Australian researchers hope an antibody found in sharks may help treat an incurable lung disease.

Idiopathic pulmonary fibrosis (IPF) scars lung tissue, causing breathing to become progressively harder. It kills more than 5,000 people each year in the UK alone, according to the British Lung Foundation. Researchers hope a new drug, inspired by an antibody in the blood of sharks, can begin human trials next year. The drug, AD-114, was developed by researchers at Melbourne's La Trobe University and biotechnology company AdAlta.

Initial testing successfully targeted fibrosis-causing cells by creating a human protein that imitated the shark's antibody, according to Dr Mick Foley, from the La Trobe Institute for Molecular Science. "Fibrosis is the end result of a lot of different insults and injuries," he told the BBC. "This molecule can kill the cells that cause fibrosis."

Debilitating symptoms

IPF symptoms include shortness of breath, especially during exercise, which gradually gets worse, and a persistent dry cough. There is currently no cure so treatment focuses on trying to relieve symptoms and slow its progression. The US Food and Drug Administration this month designated AD-114 an "orphan drug" - a move which gives tax breaks to companies attempting to find treatments for diseases.

A wobbegong shark

Dr Foley, who is also AdAlta's chief scientific officer, said the company had raised A$10 million (£6m; $7.5m) since being listed on the Australian Stock Exchange in August. It intends to use the money to take the drug to human trials in 2018. AD-114 does not involve injecting shark blood, which the human body would reject, Dr Foley said.

Other potential uses

In laboratory tests, the drug also showed potential to treat other forms of fibrosis. This included, for example, people suffering from liver disease and age-related eyesight degeneration, Dr Foley said. He added no sharks had been harmed in the process. A single blood sample was extracted from a wobbegong shark at Melbourne Aquarium, ."It would be very nice to say one day that 'this person is alive because of what the sharks told us,'" Dr Foley said.

http://www.bbc.co.uk/news/world-australia-38792405