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What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis

Rare diseases in childhood can be debilitating and require lifelong care. Since 1983, the Orphan Drug Act incentives have stimulated the development and significantly improved the availability of treatment products for patients with rare diseases.

We report an increasing pediatric orphan product designations and approvals from 2000 to 2009. The trend indicates that the Orphan Drug Act has continued to address this important unmet need. (Read the full article)




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Pediatric Orphan Drug Indications: 2010-2018

BACKGROUND:

Orphan drug development is crucial for children, who are disproportionately affected by rare diseases. Data are lacking on the number, nature, and benefit of recently approved pediatric orphan indications.

METHODS:

We classified the 402 orphan indications the US Food and Drug Administration approved between 2010 and 2018 as "pediatric" if they were approved for children only or targeted pediatric diseases. We determined the number of unique diseases targeted by pediatric orphan indications and calculated the proportion that were for (1) novel drugs, (2) non-novel drugs approved to treat ≥1 common disease, and (3) non-novel drugs approved only to treat rare diseases. Among pediatric orphan indications eligible for US Food and Drug Administration breakthrough designation (granted to drugs potentially representing major therapeutic advances), we calculated the proportion receiving this designation.

RESULTS:

Of the 402 orphan indications, 136 (33.8%) were pediatric. These 136 indications targeted 87 unique diseases; 21 diseases were targeted by ≥1 indication. Of the 136 pediatric orphan indications, 60 (44.1%) were for novel drugs, 45 (33.1%) were for non-novel drugs approved to treat ≥1 common disease, and 31 (22.8%) were for non-novel drugs approved only to treat rare diseases. Among 97 indications eligible for breakthrough designation, 20 (20.6%) received this designation.

CONCLUSIONS:

Recent orphan drug development has increased the availability of treatments for pediatric rare diseases. Most pediatric orphan indications expanded use of existing drugs, and many targeted the same disease. Some indications may represent breakthroughs, but substantial unmet need for treatments remains for most pediatric rare diseases.




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COVID-19 leading to shortage of orphan drugs in Russia

The ongoing spread of COVID-19 in Russia is leading to shortages of drugs against orphan diseases in…



  • Anti-virals/Coronavirus/Focus On/From our correspondent/Hydroxychloroquine/In Depth/Pharmaceutical/Public health/Rare diseases/Russia/Russian market


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Owlet orphaned by fallen tree gets adopted by new family (video)

Here's what happens when kind humans and open-armed owls encounter an orphaned owlet.




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Coronavirus Lockdown: Nagpur Police steps in as orphan bride's family, wins hearts on Twitter

In a heartwarming gesture, Nagpur Police stepped up to stand in as a orphan bride’s family on her wedding day after her relatives could not attend the ceremony due to the restrictions imposed as part of the nationwide lockdown due to the coronavirus outbreak.

The officers of the police department showed up for the wedding to bless the couple so that the bride does not miss her family on her special day. They shared a post about the moment on Twitter with the photo of the newlyweds with officers. The post said that the bride’s parents had died and her relatives could not attend the wedding due moment restrictions.

“The bride's parents had passed away. There was no one from her family to attend her marriage due to movement restrictions. #NagpurPolice tried to fulfill this absence.PI and staff were present to bless the newly wedded couple at #Nagpur,” said the tweet by the police, with the hashtag #alwaysthereforyou.

The post that went viral on Wednesday garnered 757 likes and was retweeted 81 times and counting. Users commenting on the post praised the Nagpur police for their noble deed.

What do you think about the post?

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Australian mum and daughter are making pouches for baby joeys and bats orphaned by bushfires

An estimated one billion animals have been killed as massive bushfires ravage across Australia.




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Mercenaries could be drafted in to extract three British orphans from Syria

Initial plans to use UK special forces to repatriate the children of a dead Islamic State fighter were met with strong resistance from the Cabinet, prompting a Foreign Office rethink.




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British children orphaned after their family travelled to Syria back in the UK with relatives

The judge, who has looked at issues relating to the children's welfare at private hearings in the Family Division of the High Court in London, revealed detail of their return in a ruling published today.




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Orphan raised by drug traffickers was part of Australia's biggest ever heroin importation

Singapore-based drug smuggler Wee Quay Tan, 48, who also has a large tattoo of a dragon inked over his shoulders and chest, found himself in the middle of the biggest drug bust in Australian history.




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Proud 'foster mother' orangutan begins new life in wild with her adopted orphaned infant

Monti and three-year-old infant Anggun, who were both rescued from human captivity, were released into the jungle of Bukit Baka Bukit Raya National Park in West Borneo earlier this month.




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Orphaned puppy is rescued from the rubble of ISIS leader's compound

A puppy (pictured) orphaned by the destruction of the Syrian compound which housed abu Bakr al-Baghdadi has been rescued after being found next to its dead mother.




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Unwilling parents, unwary orphans


In Anantapur, farm suicides are fewer than they were in 2002. But they still happen and could rise again in this fragile region. As elsewhere, agriculture is plagued by uncertainty, writes P Sainath.




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Orphans of our society


Ignored by the government, shunned by society and caught in a time-warp of their own, the nomadic castes and tribes of India are almost "non citizens" of the land. R. Akhileshwari describes the abysmal plight of such people from Andhra Pradesh and highlights the injustice and neglect that they are subject to.




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Orphans of Williamsburg

Historian Cathy Hellier describes the poignant histories of children left without parents. The question of providing for colonial orphans was split between the courts and the children’s caretakers. Listen this week to learn how colonial society looked after its littlest citizens.




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Design FX - How Orphan Black Creates Convincing Clones

Design FX dives into the incredible special effects of BBC America's Orphan Black. Mike Seymour breaks down how the team at Intelligent Creatures and Emmy Award nominated actress Tatiana Maslany work together to create convincing clones; with a combination of visual effects, rotoscoping, and incredible acting ability.




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Expert opinion on orphan drugs [electronic journal].

London : Informa Healthcare




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The Nociceptin/Orphanin FQ peptide receptor / editors, Mei-Chuan Ko and Girolamo Caló

Online Resource





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Young Wild West locating a lode, or, The orphans of Bowie Bar




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Young Wild West locating a lode, or, The orphans of Bowie Bar




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Bill Graham’s American Journey: A Rock & Roll Mastermind Started Life as an Orphaned World War II Refugee

Bill Graham once said he didn’t remember much about his childhood. Maybe that’s because he preferred to forget. One of the most influential rock & roll promoters of all time, Graham was quite literally the man behind the music. The manager of the legendary Fillmore Auditorium in San Francisco and the Fillmore East in New...

The post Bill Graham’s American Journey: A Rock & Roll Mastermind Started Life as an Orphaned World War II Refugee appeared first on Behind The Scenes.




orphan

Orphans of Williamsburg

Historian Cathy Hellier describes the poignant histories of children left without parents. The question of providing for colonial orphans was split between the courts and the children’s caretakers. Listen this week to learn how colonial society looked after its littlest citizens.




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UP: 3 held for ‘raping’ orphanage inmate