A new therapy for Cystic fibrosis helped a 15 years old patient use her lung again. Her life was significantly transformed as she herself mentioned it “like a butterfly out of a cocoon”.

Patients often die before their 40s as mucus clogs and damages their lungs and leaves them prone to infection.

A major trial on 1,108 patients, in the New England Journal of Medicine, showed a combination of drugs could bypass the genetic errors that cause the disease and may increase life expectancy.

Early studies have suggested that lumacaftor/ivacaftor, to be marketed under the brand name Orkambi, is safe for patients homozygous for F508del — the most common genetic mutation in cystic fibrosis — and lowers the rate of pulmonary exacerbations. The medicine could alter the microscopic machinery so they made runnier mucus.

Dr. Susanna McColley, professor of pediatrics at Northwestern University mentioned it as “groundbreaking finding” and Prof Stuart Elborn who led the European part of the trial from Queen’s University Belfast named it as the “fundamental treatment.”

Only half of people with cystic fibrosis make it into their 40s, but a 15 years old British patient has been on the drug Orkambi for three years and says it is “life changing.”

According to Medscape, in September 2016, The US Food and Drug Administration (FDA) has expanded the use of the cystic fibrosis (CF) drug Orkambi (Vertex Pharmaceuticals) to children aged 6 to 11 years who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

Orkambi has recently received the ‘Drug Discovery of the Year Award’ award from the British Pharmacological Society and the French ‘Prix Galien’ award for the most promising rare disease medicine in 2016.

For the first time, a robot operated inside the eye of a man who was going blind.

British surgeons have successfully performed the world’s first robotic operation inside the eye, potentially revolutionizing the way such conditions are treated.

The procedure was carried out at John Radcliffe hospital in Oxford, where surgeons welcomed its success.

Speaking at his follow-up visit at the Oxford eye hospital, Father Beaver said: “My sight is coming back.

“I am delighted that my surgery went so well and I feel honored to be part of this pioneering research project.”

Surgeons used sound waves to cure patient’s tremors. He had deep brain surgery without going under the knife.

Doctors at Imperial College Healthcare NHS Trust have treated the first patients using the new technique which avoids the need for invasive brain surgery.

The patient who has lived with a tremor for 20 years said:” Since the treatment I have been able to write my own name for the first time in many years and I will also be able to go back to using my right hand which will allow me to take on more painting and decorating jobs.”

Claire Bale, Head of Research Communications and Engagement at Parkinson’s UK, said: “The development of focused ultrasound techniques offers a new and promising tool for treating tremor.”

Scientists revealed that some psychosis cases are an immune disorder.

Some patients sectioned with psychotic conditions, such as schizophrenia or bipolar disorder, may actually have a treatable immune disorder, say Oxford University scientists. A study in the Lancet Psychiatry suggests up to one in 11 cases of psychosis may involve antibodies attacking parts of the brain.

A huge leap for prostate cancer treatment reached. Prostate cancer laser treatment could be a gamechanger for men.

Photodynamic therapy uses lasers and a drug made from sea bacteria to get rid of tumors.

Scientists at University College London have made what they believe is a real breakthrough using a drug derived from bacteria found at the bottom of the sea, injected into the bloodstream and activated in the prostate by laser beams.

Scientists created a mobile game to help detect the early onset of dementia.

The collaboration between Alzheimer’s research UK, Deutsche Telekom, game designers Glitchers and dementia scientists, is pioneering a massive crowd-sourced database on human spatial navigation. Over 47 million people in the world suffer from dementia; Alzheimer’s disease is the most form of dementia, accounting for 60% to 70% of cases. This disease affects parts of the brain that process visual information and deal with spatial awareness.

The breakthrough could lead to the first ever test for dementia, and has been facilitated by a mobile game. 

Sea Hero Quest tests gamers' navigational skills as they direct an old sailor on a journey to save his lost memories. Since its launch in May it has been anonymously recording the data of 2.4 million gamers for the biggest dementia study.

Results show that people's sense of direction declines as we age, and more rapidly in those with signs of dementia.

A breakthrough discovery bankrolled by 2014’s ALS ice bucket challenge: the new gene associated with the disease ALS, discovered.

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. While 10 percent of ALS is familial, meaning it's genetic, the other 90 percent of ALS cases are considered sporadic, or without a family history. However, it's very likely that genetics contribute, directly or indirectly, to a much larger percentage of ALS cases.

The newly discovered gene, NEK1, is only associated with 3% of ALS cases, but it is present in both inherited and sporadic forms of the disease, which researchers say gives them a new target for the development of possible treatments.

Dr. Shima Naghavi, Director of International Affairs