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Govt working on strategy to manufacture rare-disease drugs indigenously

With no domestic manufacturers developing drugs for people ailing with rare genetic diseases in India which is home to 8 crore such patients, the Government is working on a strategy...

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Rare Disease Diagnosis & Treatment: Rare Disease Girls Father Helps Thousands | The Better India

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Atrophy-related Protein BNIP3 May Be New Target in LOPD

The levels of BNIP3, a protein implicated in muscle fiber atrophy and increased autophagy — two features of late-onset Pompe disease (LOPD) — are increased in muscle samples from LOPD patients relative...

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Disabled Children Are Giving Up On Their Dreams,’ Ruchita’s Fight For Accessible Infrastructure At Schools

A 21-year-old fighting battle with a rare genetic disease calls for an inclusive infrastructure in schools and colleges for the disabled. Access to education for disabled students has become challenging....

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Preethu, who conquered SMA by becoming a CA, is no more

Kochi: Preethu Jayaprakash, 28, who became a Chartered Accountant by fighting the permanent disability arising out of the rare genetic disorder Spinal Muscular Atrophy (SMA), finally succumbed to her injuries...

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News Letter May 2022

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Government’s crowdfunding plan for treating patients with rare diseases yet to take off

The total amount of donations channelled to the rare diseases portal is Rs 1.18 lakh till date, Bharti Pravin Pawar, minister of state for health and family welfare, told the...

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News Letter April 2022

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Pfizer announces phase 3 trial of Duchenne muscular dystrophy treatment

Pfizer revealed plans to open sites in the U.S. for a phase 3 study evaluating an investigational mini-dystrophin gene therapy in ambulatory patients with Duchenne muscular dystrophy. The announcement comes...

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Tollywood’s young sensation Nikhil Siddhartha’s father Shyam Siddhartha passed away on Thursday due to a rare disease, Cortico Basal Degeneration.

Nikhil Siddhartha’s Father Passes Away; Actor Says ‘hope We Meet Again’ In Emotional Note Tollywood’s young sensation Nikhil Siddhartha is devastated after the demise of his father Shyam Siddhartha. His father passed away on...

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